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Archive for the ‘Gene Therapy Doctor’ Category

How immunotherapy is revolutionizing cancer care – Genetic Literacy Project

More than a century ago, in 1910, President William Howard Taft made what then seemed a bold but reasonable prediction: Within five years, he said, cancer will have been removed from the list of fatal maladies.

So, what happened?

Despite what seem like endless decades of hope, exhaustive research and unyielding effort by the worlds smartest scientists, we still have yet to find a cure or long-term treatments for cancer. But finally, we appear to be edging closer to the finish line, and immunotherapy might prove key.

For decades, the cancer treatment of choice has been chemotherapy. But, while chemotherapy can be incredibly effective at treating cancer, it takes a steep toll on human body. The side-effects of most chemotherapy treatments can be quite severe, and while the end result does often get rid of malignant cells, it also destroys plenty of healthy cells in the process.

Cancer is immensely complicated. Its not just one disease it can actually take over a hundred forms, and attack different parts of our body. Whats worse, what starts as one disease can mutate into something entirely different. Many tumors also contain more than one type of cancer cell.

Another challenge of treating cancer lies in the fact that there are great differences in patients physiologies, lifestyles, attitudes towards treatment, responses to treatment, genetic makeup and even epigenetic factors.

Cancer is as individual as the person who has it, explainsJoyce Ohm, PhD, at the Department of Cancer Genetics and Genomics at Roswell Park Cancer Institute in Buffalo, New York.

Lets say there are identical twin sisters, both with breast cancer. They may have been born with exactly the same genetic mutations, but one responds to therapy and one doesnt. One may live and one may die.

Its exceptionally difficult to find something that will work on everyone. Immunotherapy seeks to resolve this issue by personalizing treatments for each patient.

Immunotherapy isnt a new treatment by any means; scientists have been researching it for many years, and theyve invested a lot of time creating the right procedures and improving chances for all cancer patients.

The talk of immunotherapy started way back in 1890s, when William Coley, a physician, started researching how our immune system responds to viral infections. He hypothesized that scientists could jumpstart our natural immune response to cancer by provoking it with a controlled virus infection. But for years, little practical progress was made, and immunotherapy was viewed as having limited potential.

As crude as this method sounds, its basics eventually led scientists to explore how our own immune system responds to cancer and what can be done to target it without damaging other somatic cells. It also works on many types of cancers, even some that do not respond to chemotherapy or radiation.

Although immunotherapy is not yet as widely used assurgery,chemotherapy, orradiation therapy, immunotherapy drugs have been approved to treat many types of cancer. The one the doctor decides to use depends on the type of cancer they are tackling.

The main aim of immunotherapy today is to help activate dormant T-cells and help the immune system better recognize cancerous cells and get rid of them safely. T-cell transfer therapy basically attempts to re-engineer our immune response. Its a complicated process but so far it has shown great success. Other cancer immunotherapy treatments include immune checkpoint inhibitors that block certain chemicals in our body from stopping immune response to cancer cells; the use of cytokines, laboratory-made versions of a type of natural protein that boosts our immune response; lab-made monoclonal antibodies that bind to specific targets on cancer cells; and treatment vaccines.

The key in developing immunotherapies is finding the right cancer targets. Chow Kwan Ting, a researcher from City University of Hong Kong (CityU), who has won the famous Croucher Innovation Award in recognition of her scientific achievements, focuses on the role of plasmacytoid dendritic cells (pDCs) in cancer treatment.

These cells havent been researched thoroughly before, but theyre known to play an important part of immune systems response to viral infections. Dr. Chows studies have shown that pDCs play a role in other types of infections as well, and that they could potentially do more than simply fight off an infection: they might actually play a role in cancer immunity.

Other scientists are advancing a range of promising immunotherapies that researchers hope will lead to breakthrough cures. A team of researchers at the German Cancer Research Center and the Berlin Institute of Health are targeting the metabolic enzyme IL4I1 (Interleukin-4-Induced-1). The survival probability of patients with gliomas, a type of malignant brain tumor, decreased when the enzyme was present in higher concentrations in these tumors.

In 2004, Sophie Lucas, a researcher at the University of Louvain de Duve Institute, began studying the blocking of immune defenses in tumors in order to understand the functioning of cells that are said to be immunosuppressive (they block the bodys immune responses). The goal was to identify and remove them, thus stimulating antibodies to act against the tumor.Last August, Nature Communications published the results of the first tests carried out by Dr. Lucas and her team on a tool using what are called anti-GARP antibodies that prevent the bodys natural immune response from being blocked. It worked on mice; human studies are next.

Of course, more research is needed to turn immunotherapy research into potential cures, but the very fact that scientists keep learning new things about cancer treatment is encouraging. Scientists are looking into liver and breast cancer as they are more prevalent in Hong Kong than other types of cancer.

Immunotherapy can sometimes have similar side-effects as chemotherapy, such as nausea, vomiting and hair loss, but they are usually less severe. It can also be used in combination with radiation therapy or surgery.

Along with gene therapy and tumor DNA sequencing, immunotherapy is providing new options and helping us edge closer to promises made more than a century ago.

Claire Adams is a content creator and external associate of the City University of Hong Kong. Her goal is to promote CityU young scholars research papers that are closely related to the healthcare industry. She wishes to emphasise the importance of the research paper on rare cells and the innovative immunotherapeutic strategy, which truly brings hope to new cancer immunotherapy and vaccine. By promoting this work among the scientific and healthcare community, Claire is hoping to raise awareness of the City University of Hong Kongs contribution to society. Follow her on Twitter@adamsnclaire

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How immunotherapy is revolutionizing cancer care - Genetic Literacy Project

The good news about breast cancer – The Gazette

By Maggie Ireland, for The Gazette

October as Breast Cancer Awareness Month shines a spotlight on the battle against breast cancer and the developments in diagnosing and treating the disease that inspire hope for the future.

Breast cancer remains very common. Its still the most common cancer in women, said Dr. Ingrid Lizarraga, a clinical associate professor of surgery and breast surgeon at the University of Iowa Hospitals and Clinics in Iowa City.

But it is no longer the most common cause of death for women it is now the second most common cause of cancer death for women after lung cancer. We know a lot

more about breast cancer than we ever did before. The vast majority of women who have breast cancer will do great.

Admittedly, a breast cancer prognosis largely depends on the type of breast cancer you have and the stage at which it is diagnosed. The good news is that the prognosis has improved considerably over the years.

Mortality rates are decreasing, thanks to improved screenings and improved treatment, said Dr. Rasa Buntinas, an oncologist at PCI in Cedar Rapids. At our cancer center, 80 percent of women are diagnosed at an early stage stage 0 or stage 1 and that means a better chance of survival.

More screening options have become available in recent years.

Mammograms are still what we recommend, Lizarraga said. 3-D mammograms have been around for about five years and can be really helpful for women with dense


breasts. The recognition that breast density affects how you screen is important. Whole breast ultrasounds can also be useful.

For women with abnormal findings, a breast MRI is an option.

Discussing your personal risk factors with your doctor is the best way to determine what screenings are best for you.

The latest developments in genetic testing can further pinpoint specific risk factors.

Weve identified a set of genes that puts you at high risk for developing breast cancer, and we can test for this gene, Lizarraga said. The trick is to find those at risk who need the genetic testing. If you have concerns, talk to your doctor so they can assess your risk and give you better guidance on a screening regimen.

Today, the widely accepted recommendation is for most women to begin getting an annual mammogram at age 40.

If you have any risk factors at all, start at 40. But the latest you should start is at age 45. And you should screen every year, Lizarraga said. When youre younger, the odds are lower, but the stakes are higher.

In the past, all women were encouraged to do a monthly self-exam, but thats no longer the case.

Self-exams are no longer recommended, Buntinas said. They tended to lead to more testing and biopsies but really didnt improve outcomes. The recommendation now

is breast self-awareness. Be aware of how your breasts look and feel, and report changes to your doctor.

Lizarraga agrees that a woman should trust her instincts if she senses something is wrong.

You know your body better than anyone else, she said. It doesnt just have to be a lump it could be a different appearance of the skin or the nipple. Pay attention to how you feel and how you look, and advocate for yourself if you notice something.

Many developments have been made in treating breast cancer, particularly metastatic breast cancer, Buntinas said.

Were really getting a much better understanding of tumor biology, she said. Specific targets are being identified to better improve treatments and provide more of a personalized medicine approach. In practice, these treatments are really improving survival.

Its an exciting time in the treatment of breast cancer, Lizarraga said, because things pretty much change every day.

If a woman needs a mastectomy, we can give you a result that looks much closer to what your native breast looks like, she said. The attitude used to be that you shouldnt worry about how it looks, because you should be grateful you dont have cancer, but thats not accepted anymore.

Advancements also are being made in addressing the side effects of treatment, like lymphedema, the swelling of a womans arm or hand that can follow the removal of lymph nodes.

Weve gotten better at figuring out more effective techniques that result in less lymphedema, Lizarraga said. Were also now better able to save a womans breasts if she prefers to do that.

The biggest advances in treatment, however, have been in systemic therapies, or the use of drugs to reach cancer cells anywhere in the body, she said.

Almost all women with breast cancer will get some form of systemic treatment pills or therapy.


Weve become increasingly targeted with treatment, Lizarraga said. We start by figuring out which type of breast cancer you have. We now have genomic tests, so we can test the DNA of the tumor itself to see how likely it is to recur. That actually allows us to figure out whether a woman will benefit from chemotherapy or not.

Thats huge because we can limit the toxic treatment when its not going to be beneficial.

Treatment is more targeted than ever.

We match severity of treatment to the severity of disease, Lizarraga said. Weve figured out how to make things more personal in a lot of cases, weve changed the timing of treatment. In the past, you often came in, had surgery and then began the next form of treatment, like chemotherapy or pills.

Now, we often provide treatment before surgery and then personalize what is needed next.

Lizarraga said women of all ages need to know breast cancer is common but treatable, as long as its caught early.

If you do have a family history, be sure to get evaluated to see if genetic testing is appropriate, she said.

And know that who treats you is important, too.

Get a second opinion make sure the person youre seeing is someone who knows specifically about breast cancer treatment and the latest developments. Advocate for


Both doctors agree the prognosis for breast cancer patients is brighter than ever.

The outlook is very positive and hopeful for the future, Buntinas said.

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The good news about breast cancer - The Gazette

Harris County parents win insurance appeal for 8-year-olds MD treatment – WRBL

HARRIS COUNTY, Ga. (WRBL) A Harris County family is celebrating overcoming a major obstacle in treating their 8-year-old-son who has a rare form of Muscular Dystrophy, thanks to a village of supporters and a phone call from the Chief Medical Officer of a major insurance company.

8-year-old Jacob Saalman is like most kids. He loves being outdoors, riding four-wheelers, and playing baseball. This Halloween, he carved pumpkins with his family. Jacob loves his dad, a former Fort Benning Ranger, and his mom. Jacob adores his four-year-old brother, Hudson.

Jacob is very protective of Hudson. Hes a mother hen. Oftentimes I have to tell Jacob to stop parenting, shared Ryan and Brooke Saalman, Jacobs parents.

However, Jacob is not like most kids when it comes to his health. Jacobs handled more medical procedures, biopsies, and tests than many adults. Its heartbreaking. Jacobs mom and dad say hes resilient with a quiet strength.

I would say he is very strong and adaptable. All the things he has to do, all the medical stuff he has been through, pokes and prods and port placement and muscle biopsies. He has been through a lot, said Brooke.

Jacob is very sick. So is his brother Hudson. Both boys have Duchenne Muscular Dystrophy, a rare genetic disease, preventing their bodies from making a protein called Dystrophin that protects our muscles from breaking down with use.

Jacob plays ball now. By 12, he will most likely need a wheelchair. After that, the prognosis remains bleak for the brothers and the parents who love them.

The life expectancy is upper 20s and into the 30s. But you are not walking around or living a normal life. You are most likely on a ventilator and having a parent care of you 24/7, shared Ryan.

Jacobs medical team is convinced an FDA approved home infusion called Exondys 51 will significantly improve Jacobs quality of life by helping him produce partial Dystrophin proteins.

Its not a cure. It slows the rate of the disease, and also its been proven to help kids walk longer, and it is also shown to improve pulmonary function, and thats the issue later on down that causes a lot of problems and even death from this disease, said Brooke.

The Saalmans goal is to stave off the disease for as long as possible. They believe Exondys 51 can help keep Jacob as healthy as possible until a gene therapy is ready.

We still have hope. What gives us the most hope is there are gene therapy trials in phase three. So we are hoping within the next two or three years we will be able to have a gene therapy that will make this a much milder disease, said Brooke.

One of Jacobs physicians, Han C. Phan, MD, with Rare Disease Research LLC in Atlanta, Georgia, shared this statement with News 3:

Jacob has Duchenne Muscular Dystrophy, a slowly progressive condition for which without treatment could limit his life expectancy. Every child with DMD should receive the treatment he deserves and without such could be detrimental to his overall health.

The family says Horizon Blue Cross Blue Shield of New Jersey had twice denied Jacob Exondys 51, despite other major insurance companies approving similar patients treatment. The drug costs 300,000 a year. The company listed several reasons for denying coverage for the medicine in a letter sent to the Saalman family.

Ryan Saalman shared the familes heartbreak and frustration on his Facebook page earlier in the week. The status update was shared several thousand times and sent to News 3.

Monday, the Sallman family spoke with News 3 about their struggle since April to get the medication approved with their insurance company.

Its just frustrating to know there is a child that has this need, and we pay good money for our insurance. You think that if a doctor says this is medically necessary that they will get the medication, and thats not the case. These kids deserve that. Everything they go through. They deserve some help, said Brooke.

News 3 reached out to Horizon Blue Cross Blue Shield of New Jersey on Monday. Tuesday, News 3 worked with The Saalman family and Horizon BCBS of New Jersey to get the necessary medical privacy forms filled out to get information on the insurance companys case. However, before the process was complete, The Saalman family says they got a phone call from the insurance companys Chief Medical Officer. The family was thrilled to learn Exondys 51 has been approved for Jacob.

Today, I think we truly saw the power of community. In this fight against Duchenne Muscular Dystrophy, it takes a village, and I think our village got really big really fast. Our new village gave an 8-year-old boy a loud enough voice that Horizon BCBS has overturned last weeks denial. Jacob will be able to get the medication he needs. The Saalman Family wants to thank the thousands of people that shared our story; we truly believe it is because of all of you that this has happened, said Ryan.

The Saalman family is overjoyed. Time is not on their side as this disease doesnt slow down. With each delay comes another day Duchenne Muscular Dystrophy robs Jacobs muscles of their ability to function. The family hopes the newly approved infusion will be ready for Jacob within a few weeks. The treatment should help slow the diseases progression down and give the family the time they need to wait for a promising gene therapy they pray is on the horizon.

The day before The Saalman family learned the medication had been approved, Jacob hit the winning RBI for the final game of his baseball season in a tie game with two outs. Jacob got the game ball in the final game of what may be his last season of baseball.

And sometimes God shows up in the last game, in the last inning, with two outs and two strikes on the board, when the hope of a win is almost gone and He crushes our expectations. Man, did I ever need that reminder tonight, and maybe you do too, shared Jacobs father on Facebook.

For more information on Jacob and Hudsons battle with Duchenne MD and ways you can help, you can visit their blog: Saalman Strong. The family has been encouraged to establish a GoFundMe page: Jacob and Hudsons fight against Duchenne MD

News 3 will keep you updated on the Saalman family and the journey with their sons Jacob and Hudson.

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Harris County parents win insurance appeal for 8-year-olds MD treatment - WRBL

Biohack the ageing process by taking care of this little protein in your body – Lifestyle Asia

In 2009, studies on telomeres and telomerase (its corresponding enzyme) won three American researchers the Nobel Prize in Physiology or Medicine. Since then there has been a flurry of work surrounding this seemingly redundant protein, that has now been touted as the secret to biohacking the ageing process.

We sit down with Functional Medicine Specialist, Miles Price, to figure out why this little protein is so important to our longevity and our health.

Telomeres are proteins called nucleotides which attach to the ends of DNA strandsto protect them from damage every time the cells divide. They play a central role in the cell fate and ageing. Each time the cell divides, some of the telomere sequence gets lost so the telomere cap shrinks. Eventually, when the telomere becomes too short, the cell will either self-destruct through apoptosis or go into senescence. Senescence is an arrested cell state meaning the cell is no longer functioning at100 percent. This contributes to ageing and puts the body at greater risk of developing abnormal cells like cancer.

Getting older is a fact of life. However, the rate of how fast we age is controlled, amongst other things, by our telomere length. These bits of proteins protect our DNA from damage every time the cells divide. Telomere length generally shortens with age however we can slow down the shortening largely with lifestyle interventions. Things which accelerate the shortening include smoking, pollution exposure, lack of physical activity, obesity, stress and inflammation. Skin ageing is also affected by shortened telomeres, however the efficacy for maintaining skin health with telomerase interventions is still being investigated.

Telomere length is greatly influenced by our environment and lifestyle. Through epigenetic influences we can help protect our telomeres by doing the following:

1. Caloric restriction time-restricted eating or fasting up to a minimum of three days(with supervision).2. Evidence suggests that dietary alterations can affect our telomeres. An example ofthis is restricting protein intake.3. Take regular exercise, preferably high intensity intermittent training.4. Increase antioxidants in the diet, typically from plant foods.

A recent gene therapy study on mice consisted of inducing telomerase, the enzyme which slows down the biological clock by extending telomere length, was successful in increasing the lifespan by a further 24 percent. Whilst human trials are still a ways off, what we can do is consider some supplemental interventions together with lifestyle measures discussed earlier. NAD+ (Nicotinamide adenine dinucleotide) is a supplement which is a derivative of Niacin (vitamin B3), and it directly influences the lifespan of the cells and the way the cell ages. This is done by interacting with proteins inside the cell called sirtuins. Sirtuins stimulate various enzymatic processes inside the mitochondria and nucleus which help to protect the DNA and RNA from damage as the cell divides. As we age, our NAD+ levels decline and thus the enzymatic activity of sirtuins declines also, therefore it is becoming increasingly important to consider supplementing with NAD+ as we age.

As you age your immune system becomes weaker. How this affects you is dependent on many factors, like your diet, exercise levels, sleep patterns, and stress levels, for example. Typically our immune systems become slower to respond, the body heals more slowly from infections, and the ability of the immune system to detect and correct dysfunctional cells also declines. Key immune system organs play a role in this decline. The thymus gland located in the middle of the chest plays a central rolein activating certain white blood cells to identify invaders or rogue cells. It shrinks with age due to free radical damage.

This can be minimised by taking extra zinc and other antioxidants which protect against its shrinkage. Other influences on your immunity include caloric restriction. Research has demonstrated that if you fast for more than three days your immune system is given a real boost, by stimulating autophagy. This is the process whereby the body seeks out and removes old and senescent cells, thereby reversing ageing in that short period of time. Caloric restriction for longer than 24 hours is best supervised by a doctor or functional medicine specialist who can monitor any adverse reactions you may have.

You can test your telomere length by conducting a blood test which isolates our DNA and measures telomere length using the qPCR method. This is the most popular method of assessment. One such test is the ImmniGen Test. ImmuniGen is an at-home telomere test that assesses your immune strength and resilience. By measuring your telomere length (a biomarker of ageing), it can measure how yourimmune system is ageing, known as immunosenescence, as well as key genes that affect ageing and immune health. As telomere length is heavily influenced by our lifestyle, it is recommended to repeat the test at regular intervals to track your ageing progress.

Visit LifeHub to learn more about biohacking your telomeres for a healthier and longer life. LifeHub is currently offering ImmuniGen Testing at HK$1,280, which can be ordered online and delivered straight to your door.

LifeHub is a Modern Wellness Lab in Hong Kong offering the antidote to your busy city lifestyle. As leaders in detox and anti-ageing, we cleanse you of everyday toxins and recharge your body for better performance in work and play.

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Biohack the ageing process by taking care of this little protein in your body - Lifestyle Asia

‘I have faith and I prayed a lot’ – News from southeastern Connecticut –

Yulonda Wilbur remembers precisely the sting of when she was told she had breast cancer.

The news came after a routine physical where her general practitioner did a manual exam, and Wilbur flinched when the doctor touched a tender spot on her left breast. She was already scheduled for her annual mammogram, and her physician added an ultrasound. Wilbur remembers one of the technicians when she had the tests being initially cheerful, and then solemn, and later, the radiologist telling her he didnt like what he was seeing and shed need to undergo a biopsy.

But still, when the call came on Feb. 16, 2019, with news that she had triple-negative breast cancer a cancer that tests negative for estrogen receptors, progesterone receptors and excess HER2 protein and therefore does not respond to normal hormonal therapy medicines or those that target HER2 protein receptors her world metaphorically fell apart.

It was just that moment that changes your life forever, said Wilbur, now 53 and back at work since early this year as a patient care assistant at Lawrence + Memorial Hospital, where she has been employed for two decades.

That journey from her initial physical in December 2018 would include a lumpectomy, followed by chemotherapy, then an additional diagnosis of DCIS, or cancer in her milk ducts, and finally a mastectomy in October 2019. This year has been rough with COVID, but for Wilbur, a mother of four grown children, 2019 was the year she battled breast cancer and shes grateful she didnt wage that war during a pandemic.

While shes worked at L+M for 20 years, shes been a patient care assistant for more than three decades and said working in the medical field doesnt make a breast cancer diagnosis any easier.

What shed learn along the way is that theres a higher prevalence of triple-negative breast cancer in African-American women and that friends and family are essential to get you through.

My advice is dont go it alone. Ask for help. You need support, said Wilbur, who lives in Waterford. And ask questions if you dont understand.

After the diagnosis, when one physician came into the room where Wilbur was waiting and started talking at her, Wilbur reminded her that she hadnt introduced herself and asked her to slow down and explain things more thoroughly.

You need to understand what you are going through, and it is a lot to take in, she said.

The lumpectomy was painful, the chemotherapy made her nauseous and left a distasteful film on her tongue, and she developed neuropathy, anemia and fatigue.

I heard a sermon once, said Wilbur, who is active in her church. And what they said was, When you say why me, what you are really saying is why not someone else? So I just accepted it for the most part, and I wasnt happy about it, but I accepted it.

Shes an advocate of regular checkups and mammograms, of all preventive medical care, and marvels that someone who comes from strong stock, like she does, ended up so ill.

Im one of those people who dont get colds, who dont get sick, but I did, she said. There was no history of breast cancer in her family, and shed test negative for the BRCA gene.

When Wilbur was invited to attend a breast cancer support group at Smilow Cancer Hospital Care Center in Waterford, she initially resisted.

I didnt want to sit around with people who were angry, or be depressed, she said. But it wasnt that way, everybody takes a different path, everyone has their own journey.

Not so long ago, when Wilbur found a lump on her right breast, she panicked, but the diagnosis was a benign cyst.

Its not that I cant, but I dont want to go through it again, she said. Later, I thought maybe I should have had both breasts removed.

Shes back at work at L+M and doing the things she enjoys walking, reading and keeping active in her church.

I have faith, and I prayed a lot when I was going through it," she said. "I asked Jesus to get me through it, and He did."

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'I have faith and I prayed a lot' - News from southeastern Connecticut -

Piqray Improves Overall Survival for Advanced-Stage Hormone-Receptor-Positive, HER2-Negative Breast Cancer With PIK3CA Mutation –

Postmenopausal women and men with advanced-stage hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation that grew during or after hormonal therapy lived about 8 months longer when treated with the combination of Piqray (chemical name: alpelisib) and Faslodex (chemical name: fulvestrant) compared to Faslodex alone.

These latest results from the SOLAR-1 study were presented on Sept. 19, 2020, at the European Society for Medical Oncology Virtual Congress 2020. Read the abstract of Overall survival (os) results from SOLAR-1, a phase III study of alpelisib (ALP) + fulvestrant (FUL) for hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2) advanced breast cancer (ABC).

Advanced-stage breast cancer is breast cancer that has spread to tissue near the breast (locally advanced) or to parts of the body away from the breast, such as the bones or liver (metastatic).

Overall survival is how long a person lives, whether or not the cancer grows.

Piqray is a targeted therapy medicine that inhibits the PI3K pathway. The PI3K pathway helps all cells both healthy and cancer cells get the energy they need. When this pathway is overactivated because of a mutation in the PIK3CA gene, it can allow cancer cells to survive and grow. PI3K inhibitors block this pathway, with the goal of killing cancer cells.

In hormone-receptor-positive breast cancers, a PIK3CA mutation also can make the cancer resistant to hormonal therapy medicines.

About 40% of people diagnosed with hormone-receptor-positive, HER2-negative breast cancer have a PIK3CA mutation.

Piqray is a pill taken by mouth.

Faslodex is a hormonal therapy medicine called an estrogen receptor downregulator. Faslodex sits in the estrogen receptor in breast cells so the cell cant receive estrogens signals to grow and multiply. Faslodex also reduces the number of estrogen receptors and changes the shape of breast cell estrogen receptors so they dont work as well. Faslodex is a liquid that is given once a month as an injection into a muscle.

The SOLAR-1 trial included 572 people diagnosed with advanced-stage hormone-receptor-positive, HER2-negative breast cancer that had previously been treated with hormonal therapy; 341 of the cancers had a PIK3CA mutation. The people in the study were either postmenopausal women or men.

The people were randomly split into two treatment groups:

Median follow-up time was 30.8 months. This means that researchers tracked half the people for a longer time and half the people for a shorter time.

For the 341 people diagnosed with cancer with a PIK3CA mutation, overall survival was:

This difference of about 8 months was not statistically significant, which means that it could have been due to chance and not because of the difference in treatment.

Still, the researchers who did the study said the difference in overall survival was clinically meaningful and supports using Piqray to treat advanced-stage hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation that has grown after hormonal therapy.

Like almost all cancer medicines, Piqray can cause side effects, some of them severe.

The most common side effects of Piqray are:

Piqray also may cause serious side effects, including:

If youre a postmenopausal woman or a man who has been diagnosed with advanced-stage hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation that has grown after treatment with hormonal therapy, the latest results from the SOLAR-1 study are very encouraging.

Earlier results from the SOLAR-1 trial showed that Piqray and Faslodex led to longer progression-free survival compared to Faslodex alone. Progression-free survival is how long a person lives without the cancer growing.

These latest results showing 8 months longer overall survival for people treated with Piqray and Faslodex, while not statistically significant, offer more support for using Piqray to treat this type of breast cancer.

If youre being treated for advanced-stage hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation that has stopped responding to the first hormonal therapy, you and your doctor are likely considering several treatment options, including Piqray. Together, you will weigh the benefits and risks of each treatment and decide on the best treatment plan for your unique situation.

Written by: Jamie DePolo, senior editor

Published on October 16, 2020 at 11:36 AM

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Piqray Improves Overall Survival for Advanced-Stage Hormone-Receptor-Positive, HER2-Negative Breast Cancer With PIK3CA Mutation -

Beating Cancer: Prevention must be ‘cornerstone’ of EU’s new approach – The Parliament Magazine

The European Parliament voted by a large majority to set up three special committees and a committee of inquiry for 12 months, along with a permanent subcommittee. Of those three special committees, one is the Special Committee on Beating Cancer, which is tasked with looking into different ways and opportunities for the EU to take specificaction, revise legislation and other compelling measures in order to prevent or to fight cancer.

Across the European Union, cancer is the second leading cause of death after cardiovascular disease, claiming the lives of 1.4 million Europeans each year and causing the loss of 2.3 million potential working years due to premature deaths as well as a 70bn loss in productivity.

The burden of cancer brings unimaginable suffering for patients, survivors and caregivers alike, all while stretching available healthcare resources. Cancer is an age-old disease which we are only now beginning to understand, thanks to major breakthroughs over recent decades.

We know that cancer is a complex genetic disease with more than 100 cancer types, involving nearly 1,000 known cancer-related genes, all of which points towards a small probability of ever finding a silver bullet that will treat all cancers. Our best option to beat cancer is to have a comprehensive approach, emphasising the importance of prevention and early detection, while supporting the development of novel therapies and improving follow-up care.

Our best option to beat cancer is to have a comprehensive approach, emphasising the importance of prevention and early detection, while supporting the development of novel therapies and improving follow-up care

Although this plan seems straightforward, the truth is that things are much more complicated. The 33 sitting members of the Parliaments Special Committee on Cancer will act as a bridge between stakeholders, from patients and doctors to industry and research institutes, making sure that all needs are heard and proper funding is sought. It is the Committees job, in partnership with the European Commission, to find all the legal means that will allow the Plan to Beat Cancer to take shape, setting up the political agenda and mapping its main objectives.

Fighting cancer is not an easy endeavour, nor is it cheap. Since 1971, when the US government declared war on cancer, more than $200bn has been spent by Washington alone on research and development of new medical treatments and gaining valuable knowledge on the nature of this disease.

We must grasp from the very beginning the magnitude of overcoming the colossal challenge that lies ahead. One major problem we are dealing with is the huge disparity between EU Member States in terms of cancer fighting programmes. Statistics vary widely from country to country, with more cancer survivors living in the west than in the east.

This is due in part to extensive national screening programmes, better access to specialised treatment and better health coverage. We have to ensure that European citizens enjoy the same level of health care regardless of their nationality. As any doctor will tell you, why treat when you can prevent? Between 30-50 percent of all cancers are preventable.

Prevention must be the cornerstone of our policies. We must set up the EU agenda on promoting a healthier lifestyle, emphasising the need to have a healthy diet, curbing smoking and alcohol consumption, all paired with daily exercise.

Environmental pollution and occupational carcinogens are other substantial risk factors that need to be addressed. When it comes to tumours, early diagnosis is key to improving patient outcomes. We need EU-wide screening programmes for the common types of cancer, improving coverage rates and making sure that patients have easy access to screening centres in all Member States.

We should incentivise research and development for better diagnosis equipment and integrate frontrunner technologies such as liquid biopsies and Artificial Intelligence in the process. Through developing a better understanding of how cancer works, we now have access to more efficient therapies, like personalised medicine or gene therapy.

The 33 sitting members of the Parliaments Special Committee on Cancer will act as a bridge between stakeholders, from patients and doctors to industry and research institutes, making sure that all needs are heard and proper funding is sought

But there is also a growing concern with regards to the affordability of certain treatments. Therefore reducing the cost and access of treatment for cancer patients and ensuring the sustainability of our health systems must be our top priority.

Rare and very rare types of cancer - about 20 percent of all cases - most of them being paediatric cancer, are largely overlooked by industry and research institutes. Encouraging R&D of novel treatment for rare and very rare cancers can be achieved through alternative financing mechanisms, such as milestone prizes.

By the end of our lifetimes, each one of us will have a one-in-two chance of developing a form of cancer and a one-in-five chance of dying from cancer; this is not a warning, but a reality check that most of us will have to face unless we start acting decisively.

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Beating Cancer: Prevention must be 'cornerstone' of EU's new approach - The Parliament Magazine

Watch: New 5-minute injection a game-changer in breast cancer therapy in UAE – Gulf News

Dr Humaid Al Shamsi delivers the new injection to patient Joanne Resta Image Credit: Supplied

Abu Dhabi: A brand new subcutaneous injection is set to revolutionise treatment for patients battling breast cancer.

The medicine a combination of the prescription drugs pertuzumab and trastuzumab can be delivered in a simple five-minute injection, instead of the three to five hours needed to deliver the drugs in the form of an infusion.

First time outside US

Called Phesgo, it has been licensed in the UAE and was offered on a trial basis to a patient last week at the Burjeel Cancer Institute. This represented the first time the injection was delivered outside the United States, Dr Humaid Al Shamsi, director of oncology at Burjeels parent company, VPS Healthcare, told Gulf News. The new drugs delivery coincides with Breast Cancer Awareness Month, which is marked every October.

Dr Al Shamsi, who is also president of the Emirates Oncology Society, said the quick and easy form of drug delivery will help improve patients quality of life, in addition to reducing the costs of treatment. This form of treatment should be the standard of care for cancer in the near future, he said.

Phesgo is also the only medicine of its kind available at present, which is why its availability in the UAE will be a game-changer, the doctor added.

Breast cancer type

The medicine can be used for patients who have HER2 positive breast cancer, which affects nearly 25 per cent of breast cancer patients. HER2 itself is a gene that produces HER2 proteins, or receptors. When the gene isnt working properly, it reproduces too many copies, which leads to overexpression of the HER2 protein. This causes uncontrolled breast cell division and the formation of tumors representative of HER2-positive breast cancer.

Dr Al Shamsi said Phesgo can be given in the early stages, as well as for a while in the advanced stages.

Major benefits

Typically, patients receive this combination of drugs for a year, along with chemotherapy for three to four months. Patients will still have to come in for the chemotherapy, but they can be trained to take the Phesgo injections at home. Even if they come in to the hospital to receive the injections, the shortened treatment time frees up cancer chairs, and reduces insurance costs, he explained.

In addition, patients with advanced HER2 positive breast cancer also need to take the medicine for life, so this medicine makes treatment delivery much more convenient.

Insurance approval

Dr Al Shamsi said the prescription medicine is pending insurance approval, but as soon as this is received, at least 50 of his patients would qualify to receive Phesgo. He also explained that the injection has no additional side effects, save some minor discomfort at the inject site, compared to when the injections are given as infusions.

So convenient

Joanne Resta, 42, a homemaker from the Philippines, was the first patient in the UAE to receive Phesgo, and she said it would greatly ease her treatment. I was diagnosed with breast cancer in January 2020, and have already finished the chemotherapy prescribed. I have to now do this pertuzumab-trastuzumab targeted therapy for a year, once every three weeks. Every time I have to come in to the hospital for an infusion, it takes me five to six hours, and I also have to find a sitter with whom I can leave my two children, aged four and five. So this new injection sounds wonderful to me. It is so covenient, Resta said.

Breast cancer is the most commonly detected cancer among women in the UAE, with more than a 1,000 new cases reported every year. It is also the cancer with the second highest rate of mortality in the UAE, after colorectal cancer.

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Watch: New 5-minute injection a game-changer in breast cancer therapy in UAE - Gulf News

Homer Glen Mom and Nurse Breast Cancer-Free after Radiation Therapy Clinical Trial at UChicago Medicine Comprehensive Cancer Center at Silver Cross -…

As a nurse, Joellyn Koscik knows how important regular health screenings are.

So when the Homer Glen wife and mother turned 40 last year, she followed theAmerican Cancer Societys guidelinesfor breast cancer screening and had her first mammogram.

Never in a million years did she think it would result in a cancer diagnosis.

The Unexpected Diagnosis that Changed Her Life

Kosciks story began in the spring of 2019, when radiologists flagged a possible area of concern in her right breast. Following a diagnosticmammogram,ultrasound and biopsy, her doctor delivered the news:triple negative invasive ductal carcinoma, stage 3. After completing a successful 4-month clinical trial treatment at UChicago Medicine in Hyde Park, Koscik underwent a double mastectomy with reconstruction in June 2020.

Second Clinical Trial a Precautionary Measure

Although the first round of treatment and mastectomy was successful, Kosciks treatment wasnt over. She was referred toDr. Anne McCall, M.D.,Radiation Oncologist at theUChicago Medicine Comprehensive Cancer Center at Silver Cross Hospitalin New Lenox, who immediately thought Koscik would be a great candidate for a radiation therapy study she was doing.

Joellyn was a perfect candidate for this trial because she had such a great response to her chemotherapy and a very successful reconstruction, said Dr. McCall.

In July of this year, Koscik started her second clinical trial, this one much closer to home atSilver Cross. The trial involved the use of hypofractionated radiation therapy after mastectomy and reconstruction.

Hypofractionationis a form of external beam radiation therapy that emits high-energy X-ray beams carefully aimed at the breast. Conventional radiationis delivered through the same machine, but with hypofractionation, tumors receive a higher dose of radiationper treatment session.

The point of the trial is to see the safety and effectiveness of a higher radiation dose over fewer sessions, which I received, versus a lower dose of radiation at more frequent sessions, which is the standard for radiation therapy, Koscik explains. The hope is that the radiation therapy after mastectomy will reduce my risk of the cancer returning and improve my outcomes.

Care Close to Home Made Treatment Much Easier

Over the course of three weeks in July and August, Koscik received 16 radiation therapy treatments with Dr. McCall, instead of the usual 25. The Monday-Friday sessions lasted only 30 minutes, so living just 15 minutes from theUChicago Medicine Comprehensive Cancer Center at Silver Crossmade getting to and from her treatments a breeze. Fortunately for Koscik, the side-effects were minor; fatigue and redness at the radiation site meant she could continue to work full time as a nurse, her role for the last 20 years.

During her first clinical trial, Koscik took a blood test that uses DNA analysis to identify harmful changes, or mutations, in either one of the two breast cancer susceptibility genes, BRCA1 and BRCA2. With the results of this test, called aBRCA test, Koscik discovered that even without a family history of breast cancer, she is BRCA1-positive (a genetic mutation of the BRCA1 gene), increasing her likelihood of having breast, ovarian and other reproductive organ cancers.

That news solidified her decision to undergo the mastectomy.

Since the trial only recently ended, I have an upcoming appointment with Dr. McCall to see how I did. I do plan on discussing with her the possibility of removing my ovaries and fallopian tubes as a further precautionary measure, says Koscik.

A Supportive Tribe Behind Her

Koscik lights up when talking about the people who never left her side and kept her spirits high through the toughest of days. My husband, Scott and our 11-year old son Jason have been with me through all of this, she beamed. Also, my family, my in-laws, my girlfriends, neighbors and co-workers, they never let me get down on myself and continue to encourage me on this journey.

Koscik also has many compliments on the care she received atSilver Cross Hospital, I would recommend Silver Cross to anyone. Everyone who cared for me, from the doctors and nurses, the radiation team and thelymphedema therapists, they were all so kind and treated me with love and care. During this COVID-19 pandemic, I am just so grateful to have been able to have my treatments, and Im beyond thankful for the great results Ive received so far.

To learn more about the cancer care offered at Silver Cross Hospital,

Silver Cross Hospital

1900 Silver Cross Blvd

New Lenox, IL


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Homer Glen Mom and Nurse Breast Cancer-Free after Radiation Therapy Clinical Trial at UChicago Medicine Comprehensive Cancer Center at Silver Cross -...

Teenager among first to receive gene therapy treatment for rare condition – KRDO


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Pittsburgh, PA (KDKA) A West Virginia teen is among the first in the country to get gene therapy treatment for a rare condition.

Kendra Goins has Hurler Syndrome, meaning her body cannot break down certain types of sugar. In addition to damaging her organs, the condition also makes it difficult for her to grow.

Ive spoken with experts all across the country, all the way from the chemist that made this down to the doctor who has used it. This looks like her golden ticket, said Sheryl Goins, Kendras mother.

The goal of the treatment is to force Kendras body to produce the enzymes to break down sugars.

Please note: This content carries a strict local market embargo. If you share the same market as the contributor of this article, you may not use it on any platform.

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Teenager among first to receive gene therapy treatment for rare condition - KRDO

Proposition 14: With Just Handful of Cures, California Stem Cell Agency’s Fate Is In Hands of Voters – KQED

A Yes vote authorizes the state to sell $5.5 billion in general obligation bonds primarily for stem cell research and the development of new medical treatments in California. A No vote would mean the state's stem cell research agency will probably shut down by 2023.

In the ramp-up to the 2004 election, a California TV viewer may have come across the popular actor Michael J. Fox urging her to vote Yes on a state proposition. His voice slurred faintly by Parkinsons disease, he still sounded wry, boyish and familiar.

My most important role lately is as an advocate for patients and for finding new cures for diseases, said Fox, eyes level with the camera. Californias Stem Cell Research Initiative 71 will support research to find cures for diseases that affect millions of people, including cancer, diabetes, Alzheimer's and Parkinson's.

Within that 30-second spot, Fox, diagnosed at age 29 with a neurodegenerative disorder that typically does not strike until after 60, used the word "cures" three times.

Proposition 71, which passed with 59% of the vote, authorized the sale of $3 billion in bonds to create an agency that funded stem cell research. The successful campaign grew out of a time, in the early 2000s, when the promise of stem cell and regenerative medicine excited both scientists and the public.

Whether the project has lived up to that promise is a matter of opinion. How voters view the record of the agency may go a long way in their decision whether or not to replenish the fund, which is fast running out of money, with an additional $5.5 billion to be raised with new bonds authorized by Proposition 14, now on the ballot.

President Bush A Demon to Attack

Scientists since the1800s have known about stem cells, which are not yet dedicated to any particular anatomical function and have the potential to become nerve cells, blood cells, skin cells or any other type. They are found in blastocysts, which are human embryos four to five days after fertilization, and in a few areas, such as bone marrow and gonads, in adults.

In the late 1990s, researchers developed ways to steer the development of these cells, and the possibilities for improving medicine seemed endless. If malfunctioning cells were at the root of a particular disease, could new healthy cells tailored to the job fix what was wrong? Scientists and many members of the public were eager to find out.

Anti-abortion groups, however, a key constituency of President George W. Bush, opposed the research, and in 2001 he limited federal funding to a few existing lines of embryonic stem cells, severely curtailing research.

Some in the state of California wanted to get around Bushs restrictions, and Proposition 71 was born.

"(T)hey had this demon they could attack in the campaign the Bush administration," said David Jensen, author of "California's Great Stem Cell Experiment," who also writes the blog California Stem Cell Report. "They could say, 'This is a great opportunity, and the only way we're going to get it done is to do it here in California.'"

The measure created the California Institute for Regenerative Medicine. The stem cell research agency is unique in the U.S.

"No other state has done this kind of level of funding and focus on this kind of thing, said Jensen. It's a really cutting-edge area of science."

A Few Successes

The pace of innovation has been slower than many hoped. As it turned out, grand discoveries were not around the corner, and to date there is no widespread stem cell treatment approved for the public. To date, CIRM has funded more than 64 trials directly and aided in 31 more. Not all have or will result in treatments.

But despite the lack of a marquee cure like one for Alzheimers or Parkinsons, the agency has seen some notable triumphs.

"Probably one of the most spectacular successes they have certainly so far," said Jensen, "is clinical trials that have saved the lives of what they say are 40 children."

Those children were born with severe combined immunodeficiency (SCID), commonly known as "bubble baby syndrome," a rare, generally fatal condition in which a child is born without a working immune system. An FDA-approved gene therapy that grew out of CIRM-funded research can now cure the disease by taking a patients own blood stem cells and modifying them to correct the SCID mutation. The altered cells generate new, healthy blood cells and repair the immune system.

The FDA has also approved two drugs for rare blood cancers that were developed with CIRM funds.

Sandra Dillon, a graphic designer in San Diego, credits one of the drugs with saving her life. She was diagnosed when she was just 28, in 2006. Her doctors told her they would try to manage her symptoms, but that she was going to get progressively sicker.

"Even just the idea of a cure or getting better wasn't even on the table back then," said Dillon, who is featured in ads for the Yes on 14 campaign.

"I remember just praying and begging into the universe, please, someone just look at my disease, please someone help, who is going to look at this thing.

By 2010, Dillon was extremely ill. She connected with a doctor at UC San Diego who received early-stage funding from CIRM and told her she could take part in clinical trials.

"For the first time, there was this moment of, 'Oh, my gosh! There are researchers doing something. And it could help me and I can get access to it.' It was amazing."

The drug received FDA approval in 2019, and today Dillons cancer has retreated to the point where she can live a normal life.

"I love that I am not tethered to a hospital anymore. I can go out on long backpacking trips and hiking and surfing," she said. "I am a completely different person with this drug. And I have a whole future ahead of me."

The original funding raised by Proposition 71 is running out. Proposition 14 would authorize the sale of a new bond to refill the agency piggy bank. Gov. Gavin Newsom, the UC Board of Regents, and scores of patient advocacy groups also support the measure.

Many newspaper editorial boards, however, oppose the proposition, including the San Francisco Chronicle, Mercury News and Los Angeles Times.

Right now the state still owes about $1 billion toward the debt created by Proposition 71. If Proposition 14 passes, the yearly price tag to pay off the new bond would be about $260 million per year for about 30 years.

One of the selling points of the original proposition was the potential for the state to earn big money in royalties from the treatments it helped develop, says Jeff Sheehy, an HIV patient advocate and the only CIRM board member to oppose Proposition 14.

"The promises were made that this would pay for itself. We would be able to pay back the bonds with the money we would get from royalties, etc., etc.

That has not worked out as envisioned: CIRM estimates it has received less than $500,000 in royalties. Early this year, Forty Seven, a company whose therapies were heavily funded by CIRM, sold to Gilead for $4.9 billion. While millions went to various researchers, neither CIRM nor the state of California received anything.

One of the flaws in the original measure is that we [the agency] cannot hold stock in the products that we develop," says Sheehy. "And that's because the California Constitution says that the state of California cannot, as a government entity, hold equity.

Proposition 14 makes it impossible for the state to use profits from its investment on, say, schools or other funding priorities. Instead, any royalties earned must be fed back into programs to make CIRM-funded treatments more affordable.

"What it does is it basically takes all of our returns that we get from this and gives it back to the pharmaceutical and biotech companies," said Sheehy. "It becomes just a blatant giveaway to these companies when we should be requiring access and requiring fair pricing."

Sheehy says he supports medical research, but doesn't like the state going into more debt to pay for it. The greater the state's obligations in bond money, which has to be paid back with interest, the less there is in the general fund, and Sheehy says the state has more pressing needs than stem cell research things like housing, education and transportation.

"The biggest and perhaps the most compelling reason why I feel so strongly that this is not a good idea is that we simply cannot afford it, he said. "If we think this is so important," asks Sheehy, "why don't we just don't pay for [this research] out of the general fund? It would be cheaper.

Opponents of Proposition 14 also point to longstanding complaints of conflicts of interest among the agency board. Most of the $3 billion distributed by the agency has gone to institutions with connections to board members. Critics say the structural conflicts of interest between the board and agency are not addressed in the new measure. Proposition 14 would balloon an already huge board of 29 members to 35.

Funding needs for stem cell research also are not as acute as they were back in 2004. The federal National Institutes of Health now funds some basic stem cell research, spending about $2 billion a year, with $321 million of that going toward human embryonic stem cell research. And private ventures, like nonprofits started by tech billionaires, are pouring more money into biotech.

The problem with assuming that, says Melissa King, executive director of Americans for Cures, the stem cell advocacy group behind the Yes on 14 campaign, is that CIRM fills a neglected funding need.

The NIH does not fund clinical trials at nearly the rate that CIRM can and has been, King said.

She says that's important because of what she calls the "Valley of Death," where promising early-stage research frequently fails to translate into promising treatments that can be tested in clinical-stage research. (What works well in a test tube often does not work well in an organism.) This weeding-out process is costly but necessary. And its where CIRM focused a lot of its effort.

The first- and maybe even second-phase clinical trials, its very difficult to get those funded, King said. It is too much of a risk for business to take on on its own. Venture [capital] isnt going there. Angel [funding] isnt going there.

What voters have to ask themselves, says writer Jensen, is whether stem cell funding is "a high priority for the state of California? Different people make different judgments about that."

CIRM supporters say if Prop. 14 doesn't pass, critical research will stall. Others say federal and private funding will step in and fill the gap.

Absent new funding, the institute expects it will wind down operations leading to a complete sundown in 2023.

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Proposition 14: With Just Handful of Cures, California Stem Cell Agency's Fate Is In Hands of Voters - KQED

Teenager among first to receive gene therapy treatment for rare condition – NewsChannel 3-12 – KEYT


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Pittsburgh, PA (KDKA) A West Virginia teen is among the first in the country to get gene therapy treatment for a rare condition.

Kendra Goins has Hurler Syndrome, meaning her body cannot break down certain types of sugar. In addition to damaging her organs, the condition also makes it difficult for her to grow.

Ive spoken with experts all across the country, all the way from the chemist that made this down to the doctor who has used it. This looks like her golden ticket, said Sheryl Goins, Kendras mother.

The goal of the treatment is to force Kendras body to produce the enzymes to break down sugars.

Please note: This content carries a strict local market embargo. If you share the same market as the contributor of this article, you may not use it on any platform.

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Teenager among first to receive gene therapy treatment for rare condition - NewsChannel 3-12 - KEYT

Greenbrier County teen to be among first in nation to receive gene therapy for Hurler’s Syndrome – WVVA TV

ALDERSON, W.Va. (WVVA) A Greenbrier County teen is about to become one of the first five patients in history to receive an experimental gene therapy for Hurler's Syndrome.

The Alderson native is in many ways your typical 13-year-old. "I like to help my dad feed cows....and donkeys," said Kendra Goins.

But life hasn't always been easy for Kendra. The extremely rare condition makes it impossible for her body to breakdown certain sugars. In addition to causing damage to her organs, the condition makes it difficult for her body to grow.

But whenever anyone has anything to say about it, her sister, Kiristen is always the first time stand up.

"Me with my big head is always the one to jump in," said Goins, who said she has gotten into quite a few quarrels over the issue at school in defense of her sister.

She worries though that she won't be able to jump in when Kendra heads to California soon for a clinical trial. Kendra is heading into the treatment with her first bone marrow transplant nearing the end of its course.

"I've spoken with experts across the country from the chemists who made the drug to the doctor who has used it. This looks like her golden ticket," said Kendra's mother Sheryl Goins.

Administered through the brain, the goal of the gene therapy is to help her body produce the enzymes she needs to survive.

While the cost of the clinical trial and airfare is covered, the family said they need help with expenses they will incur during their three-month stay.

To learn more about how you can help, visit

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Greenbrier County teen to be among first in nation to receive gene therapy for Hurler's Syndrome - WVVA TV

Regenxbio RGX-121 Clinical Program, And Other News: The Good, Bad And Ugly Of Biopharma – Seeking Alpha

Regenxbio announces expansion of RGX-121 clinical program

Regenxbio Inc. (RGNX) announced the expansion of its Mucopolysaccharidosis Type II clinical program. The company stated that the expansion will allow it to gain additional understanding of the neurodegenerative aspects of the disease. The expansion will also let Regenxbio in a broader patients population.

The Phase I/II study seeks to examine a single intracisternal administration of RGX-121 in patients under the age of 5 years. All these patients have severe MPS II. The company has already dosed six patients across two dose levels. The main aim of the study is to assess the safety and tolerability of RGX-121 and to evaluate the impact of the drug candidate on biomarkers of I2S enzyme activity, neurocognitive development and other clinical measures.

Regenxbio intends to start expanded enrollment of patients in Cohort 2 immediately. The decision has been taken based on the basis of recommendations made by the Independent Data Monitoring Committee and MPS II treating physicians. Steve Pakola, M.D., Chief Medical Officer of Regenxbio said, Regenxbio is committed to advancing potential gene therapy treatment options for MPS II, as there remains a significant unmet medical need to address the neurological manifestations and prevent or stabilize cognitive decline for patients.

As of September 16, 2020, RGX-121 was found to be well-tolerated in all six patients. No drug-related serious adverse events were reported. The company now plans to dose up to six additional patients with a second dose level of 6.5x1010 genome copies per gram (GC/g) of brain mass. Regenxbio expects to provide further updates from the study by the end of 2020.

Regenxbio also reported that the FDA has cleared its Investigational New Drug Application. The company plans to start the second Phase I/II trial of the drug candidate for treating pediatric patients with severe MPS II ages 5-18 years old. This multicenter, open-label trial will enroll up to six patients and the drug candidate will be administered directly to the cerebrospinal fluid through intracisternal or intracerebroventricular injection.

Regenxbio plans to open a new observational natural history trial for enrollment in the second half of 2020. This trial will study the neurocognitive development and key biomarkers in patients with severe MPS II. The trial will prospectively record the changes in neurodevelopmental issues of cognitive, behavioral and adaptive function over a period of time. It will also evaluate biomarker activity in serum, urine and the CSF. The trial will enroll up to 40 patients between the ages of 1 month and 8 years with genetic diagnosis of severe MPS II. The assessment will be carried out for up to two years.

RGX-121 is being developed for treating Mucopolysaccharidosis Type II. It aims to work by using the AAV9 vector for delivering the human iduronate-2-sulfatase gene which is responsible for encoding the iduronate-2-sulfatase (I2S) enzyme to the central nervous system. RGX-121 has been granted orphan drug product, rare pediatric disease and Fast Track designations by the FDA.

Investment Thesis: Regenxbio had a tumultuous past year and the stock has lost nearly 50 percent of its Pre-Covid valuation. While the latest news is encouraging, it may be a little too early to gauge the companys prospects ahead.

AbbVie Inc. (ABBV) announced that it has taken a voluntary decision so pull Fibristal from the Canadian market. The decision has been taken after the news of certain patients in Europe contracting rare cases of liver injury and requiring transplantation. AbbVie integrated Allergan's business to its core in May this year. The drug belongs to Allergan and the company decided that the recall is the best option in the prevailing circumstances.

Fibristal contains Ulipristal acetate as one of its main ingredients. Earlier this year, the European Medicines Agencys Pharmacovigilance Risk Committee (PRAC) had recommended the cancelation of the marketing authorization of products containing ulipristal acetate used for treating symptoms of uterine fibroids.

Fibristal in approved in Canada for treating moderate-to-severe symptoms of uterine fibroids in adult women of reproductive age. It is also approved as an intermittent treatment of moderate-to-severe signs and symptoms of uterine fibroids in adult women of reproductive age who are not eligible for surgery.

Health Canada is monitoring the effectiveness of the recall. It has advised the patients who take Fibristal to a) stop treatment and contact their doctor to discuss other treatment options; and b) immediately report to their doctor signs and symptoms of liver injury such as nausea, vomiting, stomach ache, severe tiredness, yellowing of the eyes or skin, or dark urine, which could occur after stopping treatment. Health Canada has also issued directives to healthcare professionals to stop prescribing or dispensing Fibristal and to carry out appropriate liver function monitoring.

Investment Thesis: AbbVie is a blue-chip stock with solid foundation. This negative news is not likely to have any material impact on the stock price as the company maintains its robust long-term outlook.

Ionis Pharmaceuticals Inc. (IONS) announced inking a multi-year collaboration with Genuity Science. The deal pertains to the discovery and development of innovative therapeutics across a range of up to 20 diseases. Ionis is looking to boost its presence in key segments with this collaboration and draw synergies.

Genuity Science focuses on high-quality, whole-genome sequence and deep phenotype data. Ionis plans to combine this approach with its proprietary antisense technology to expedite the drug development process. Brett P. Monia, Ph.D., chief executive officer of Ionis said, "The collaboration with Genuity Science gives us the potential to significantly increase our discovery opportunities and better understand the complexities of human diseases based on genetics."

Under the terms of the agreement, Genuity Science will be entitled to receive a combination of upfront payments and development milestones. It will also be entitled to gain product royalties. Genuity Science is a data sourcing, analytics and insights organization. It collaborates with global pharma companies to catalyze precision health services. It offers a wide range of services including high-quality sequencing and disease-specific data sourcing.

Ionis is mainly active in RNA-targeted drug discovery and development segment. The company has developed the first and only approved treatment for all patients, children and adults with spinal muscular atrophy. Its portfolio also contains the worlds first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. The company uses its antisense technology platform to boost its development pipeline. Its drug candidate Pelacarsen is in Phase 3 for cardio-renal indications. Some of its main development partners are Biogen, AstraZeneca and Novartis.

Investment Thesis: Ionis has a well-diversified development pipeline which is expected to keep its stock price buoyant. However, the price has tumbled quite a bit in the recent past, providing an exciting opportunity to build a portfolio with a long-term horizon.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Regenxbio RGX-121 Clinical Program, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

Women In Longevity Medicine And The Rise Of The Longevity Physician – Forbes

Dr. Evelyne Bischof speaking at the 2020 China-Israel Summit on Longevity Medicine

Over the past decade, we witnessed unprecedented advances in the field of biogerontology, and the massive convergence of biotechnology, information technology, AI, and medicine. And now we are witnessing the birth of a new field of longevity medicine, which integrates the latest advances in many of these fields of science and technology. My definition longevity medicine is advanced personalized preventative medicine powered by deep biomarkers of aging and longevity.

And, like in the field of AI for drug discovery, women are at the forefront of this revolution and there were precedents when we had to look for a male physician to make a conference panel more diverse.

One of the physician-scientists who stands out in this area is Dr. Evelyne Yehudit Bischof. I first got a note with a request for more information on one of our research papers from Dr. Bischof on December 30th, 2019 while in Shanghai. A request I almost ignored due to the heavy workload but accidentally I looked at her profile which was highly unusual. In brief, Evelyne is a German medical doctor with an MD from Max Planck Institute for Molecular Biology and Genetics, who interned at Columbia University, and Harvard MGH and Beth Israel Medical Deaconess, attending physician at University Hospital Basel in Switzerland, and associate professor at Shanghai University of Medicine and Health Sciences. She fluently spoke six languages including German, Russian, and Mandarin Chinese, which was quite impressive. The second time we met was at Human Longevity Inc, in San Diego when she was interviewing with one of the most influential entrepreneurs and investors in longevity biotechnology, Dr. Wei-Wu He to join HLI as a longevity physician.

Dr. Evelyne Yehudit Bischof

The longevity industry is rapidly emerging and longevity clinics are being set up in various parts of the world. So I decided to ask Eva a few questions to elucidate this new and emerging industry.

Alex: Eva, we know each other for almost a year and you do not fail to impress with your academic publications, public lectures, and clinical work. You are as close to the longevity physician as it can possibly get. Can you tell us a bit more about yourself and about the work that you are doing on the clinical side and on the research side?

Dr. Evelyne Bischof: Thank you, Alex it is an honor to be so generously introduced by a true innovator, scientist and entrepreneur, as well as a longevity KOL and allow me to revert the compliment. I am a rather globally oriented internal medicine specialist, with training and work experience in Germany, USA, Switzerland and China. For almost a decade now, I have been splitting my time between Shanghai and Basel, creating a path that allowed me to conclude my residency and fellowship, develop translational and clinical research niches and collaborators, as well as to engage actively in academic medical education. While my clinical work was mostly based in a university clinic in Basel in internal, intensive and onco-hematologic medicine wards, my scientific pursuits and academic teaching were mostly based in Shanghai, where I went along the track from a junior lecturer to an associate professor in 2016. My research focused primarily, but not exclusively, on oncology and being an internist at core on geroncology and precision medicine in general internal medicine. Geroncology is a crucial field that investigates the very much interlinked pathways of aging and tumorigenesis, leading to the epidemiological observation that age is the number one risk factor to develop cancer for all.

Both Switzerland and China are innovative hubs with strong medical and bioscientific profile, which allowed me to learn from some of the finest experts worldwide. The frequent travels and splitting my life between continents were not always easy, but - coming from a simple background of non-academic farmer and handcraft family Alongside - I will be forever grateful for all the great people I met and worked with, the abundant cultural nuances and differences I was able to learn and appreciate, the stimulating and constructive exchange and so much more in soft and hard skills, on professional and personal level. with the emergence of AI-based solutions in the clinic and with the rise of longevity medicine, my passion and efforts are now focused on these domains, while I continue my clinical practice in the university hospitals, academic lecturing at two medical schools (currently in Shanghai - due to COVID-19-related travel restrictions) and research/public speaking (globally - thanks to COVID-19-related shift to virtual communication).

Alex: Can you tell us about your perspective on the emerging field of longevity medicine starting from your own definition of the field?

Dr. Evelyne Bischof: With pleasure! My personal definition of longevity medicine is clear: it is precision medicine driven by deep aging biomarkers. Surely, the definition is succinct, but extremely deep. Precision medicine is per se an enormously complex and dynamic field, driven by multimodally mined data and their constant re-evaluation, reannotation and reiteration to provide qualitative and quantitative using AI-algorithm outputs applicable for clinical practice. Longevity medicine is a to say the next generation of precision medicine that evaluates the patient within the reference range for the patients ideal age (usually 20-30) and is looking for ways to reduce the gap between the current parameters and the parameters of maximum physical performance for the ideal age. Deep aging clocks as quantifiable, trackable and accurate biomarkers of aging and an indispensable component of longevity medicine. Without being able to actually measure the biological age and its changes due to interventions, longevity medicine cannot be performed. I strongly believe that this field of medicine will revolutionize healthcare and change the mindset of all the doctors, the policy makers, the stakeholders and above all: the patients. Allow me to add that I consider each of us as a patient we all suffer from aging! I also believe that citing Peter Diamandis in the future, if a physician wont be using A.I. in guiding diagnosis and therapy, it'll be a malpractice". This said, I would love to add that we need more passionate physicians in longevity and this can only be achieved with an appropriate educational setting, which will be inaugurated this month by Deep Longevity and collaborators.

Alex: What do you see as the most promising developments in the field of longevity medicine that can truly push the needle and add a few decades if not more to the healthy youthful life of the individual?

Dr. Evelyne Bischof: Besides of deep aging clocks and AgeMetrics, which I truly without cronyism embrace and would encourage all physicians to implement in their daily practice, I see a big potential in gene therapies, in (natural and designed) gerolytics and senolytics, as well as supplements that will show safe efficacy in combating senescence from the molecular to system level. Studies on AKG, rapamycin and metformin are already fueling this hope. Of course, all interventions will require a prior comprehensive precision health assessment and continuous monitoring. For the latter, the wearables and applications will certainly bring us even faster to an extension of a healthy and productive lifespan.

I am encouraged by the fact that there are two major developments, perpetuated by the racing speed of longevity medicine and geroscience. Number one: doctors are shifting from putting a patient on meds to putting a patient on a personalized longevity protocol that becomes a natural, integral, rewarding part of their lives. Number two: society is realizing that it is not important how old one is, but how one shows his/her own age. Remembering this allows one to make sure he or she does not become a slave of the myths about the elderly, but also to be mindful that even at an early chronological age, one might actually experience silent accelerating aging due to modifiable risk factors or pathomechanisms.

Alex: Without promoting Human Longevity Inc or Health Nucleus 100+, can you tell us what an average person with an average income can do to increase their performance and longevity?

Dr. Evelyne Bischof speaking at the 2020 China-Israel Summit on Longevity Medicine

Dr. Evelyne Bischof: This is a very valid question in fact, when it comes to reasonably boosting performance and creating a good base for longevity, one does not necessarily be wealthy. The components of the magic mixture are the well-known pillars of preventative and functional medicine: exercise, nutrition, supplements, moderation. However, longevity physicians are now able to customize the right proportions of each for a specific person, minding the biovariability, comorbidities, chronological age, but also lifestyle and preferences. In an extreme generalization, I would suggest caloric restriction via intermittent fasting to an overall healthy person, with at least an A-Z vitamin and mineral supplement, 15-30min workout at least 3 times a week, moderation in substance use to the minimum, but with permissible enjoyment, if needed (alcohol and cigarettes), a minimum of 6 hours of sleep without interruption, circadian rhythm (regular times) of sleep and food intake, no meals at night (at least 4 hours before night rest) and very importantly cognitive activities (books, foreign languages, crosswords), preferably rewarding ones so that the psychological wellbeing area is also covered. Everyone is able to use stairs as their gym, to not to eat before sleep, to choose water over other drinks, to laugh aloud to oneself and to learn text parts by heart (because decelerating psychological aging and cognitive decline are crucial aspects of healthy longevity). I recall I was always reading the ingredients and how to use? texts on tubes during shower, so as not to waste the time. My first sentence in Russian was actually the instruction of how to use a shampoo.

Dr. Evelyne Bischof speaking at a conference on aging and longevity

Alex: And if someone has nearly unlimited access to capital, what should they do?

Dr. Evelyne Bischof: I believe, as in any other business or property of this particular population, the individuals should seek good investments and insurance in relation to their health and the health of their significant surrounding (family, friends, workers etc.). The investment should involve as precise diagnostics as possible, that harnesses all cutting edge and untapped potential of the human genome, deep quantitative phenotyping, complete -omics and -ioms (e.g. microbiome, epigenomics, metabolomics, proteomics etc.), advanced imaging with radiogenomic algorithms etc. As it is a dynamic field, constantly evolving and implementing new features and/or better ways of interpretation, such diagnostic comprehensive checkups (or part of them) should be repeated regularly. The insurance part does not relate to a contracted policy, but to a complex entity of lifestyle recommendations and interventions lead by an entrusted longevity physician (basically a physician that can list and pronounce the aforementioned terms), who understands and permanently advances in the field, being able to combine human and artificial intelligence and customize an individual approach of prevention and (if needed) therapy for a specific patient. In addition, the leading physician needs to comprehend and implement the personal challenges and preferences of the patient, such as mostly disturbed wake-sleep rhythm, irregular and unhealthy social meals, acute and chronic stress exposure, irritability or fatigue etc., to create a program that will be realistic, allow the patient to remain compliant and engaged based on his/her educated informed decisions. Simply said: knowing 150 GB of a patients data, a physician of trust should be a good lead towards identification, mitigation and elimination of actionable diseases (years and decades ahead) and risk factors that curb the quantity and quality of life.

Alex: I know maybe 3-4 people like you in the world, who have an MD, are actively engaged in biomedical research, and work with some of the high-profile clients who are spoiled with the most cutting-edge medical care provided by the top medical institutions. And all of them are women. Why do we see such gender imbalance in the field?

Dr. Evelyne Bischof in the clinic

Dr. Evelyne Bischof: Again thank you very much for this encouraging statement, this time speaking on behalf of women in medicine, academia and STEM. As you know, one of my side areas of interest is the study of biological sex differences in various diseases, predominantly cancer, and ultimately also on the sex (biological) and gender (socio-cultural) variables influencing pathomechanisms, diagnostic and therapeutic decisions, resulting differing toxicities, follow up strategies and outcomes (recovery, chronification etc.). It was natural to engage in debates and develop curiosity about the gender distribution in academia in general. Recently, with an ad hoc group of collaborators from Europe, USA and China, we demonstrated in a Lancet Oncology paper that female representation at the podium, meaning as keynote speakers and scientific committees at the largest oncological conferences in China. Our data showed that China is much more inclusive, without an intensive active promotion or directives towards gender quotas. As you know, I am a big fan of this country, but this quantitative study once again showed how impressive this country is and perhaps we found one of the contributing factors for the nations booming leading role in biotech and medicine.

Overall however, there are indeed significant differences in various fields, as well as an overall underrepresentation of females in leadership and podium roles. I am happy to see that in longevity science and medicine, we have dedicated females that can unfold their passions and translate them into viable solutions that do impact the public and individual health. As always, the reasons are multifold, but perhaps the most important one is that in longevity, driven women are emerging in an inclusive environment that embraces non-discriminating and non-stigmatized diversion. In different words: the longevity field seems to embrace inclusion at the same (ultrarapid) pace as STEM and medicine are evolving. The sex and gender differences clearly allow to generate creativity and innovation it is a mutually perpetuating process. Last but not least, it is thanks to committed male mentors and collaborators that actually value D&I (diversity and inclusion) intuitively or knowingly (based on evidence that diverse teams outperform the less diverse one by over 35%). Most male KOLs in longevity, like yourself, promote and underline the importance of D&I. On a final note myself, personally, I have always remained at the unconscious side when facing a person I work with. Accountability, motivation and fairness have proven to be non-gender related in my experience as I have faced many challenges being a (previously young) female, permanent foreigner and on top of that blond. The typical situation at a round dinner table in China with 12 male professors usually ended up with us all laughing at my gambei with water being the only discrepancy from the norm.

Dr. Evelyne Bischof speaking at the 2020 China-Israel Summit on Longevity Medicine

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Women In Longevity Medicine And The Rise Of The Longevity Physician - Forbes

Timothy Ray Brown, First Patient to Be Cured of HIV, Dies of Leukemia – BioSpace

Timothy Ray Brown, dubbed the Berlin Patient, the first ever to be cured of HIV, died from cancer on September 29.

Brown was cured of HIV in 2007. He was diagnosed in 1995, and about a decade later was diagnosed with leukemia. A physician at the Free University of Berlin used a stem cell transplant from a donor with a rare genetic mutation that provided natural resistance to HIV in hopes of curing both diseases. It took two procedures but was successful, and in 2008 Brown was announced free of both HIV and leukemia.

Two years later he went public with the announcement.

I am living proof that there could be a cure for AIDS, he told Agence France-Presse (AFP) in 2012. Its very wonderful, being cured of HIV.

In 2019, a second HIV patient, Adam Castillejo, underwent a similar procedure. He was dubbed the London Patient. A UK resident diagnosed with HIV in 2003, Castillejo began antiretroviral therapy in 2012. He was later diagnosed with advanced Hodgkins lymphoma. He was treated with a stem cell transplant in 2016 after he received chemotherapy. He then continued to receive antiretroviral therapy for 16 months.

To evaluate whether the HIV-1 infection was actually in remission, he went off the usual antiretroviral therapy. After he had been in remission for 18 months, testing confirmed that his HIV viral load was undetectable.

The donors for both men carried a rare genetic mutation called CCR5-delta 32. This made these patients resistant to HIV. Castillejo is currently living HIV-free.

Brown, 54, was born in the U.S. He was diagnosed with HIV in 1995 while living in Berlin. He developed acute myeloid leukemia in 2007.

The leukemia that eventually led to his HIV cure returned this year, where it metastasized to his brain and spinal cord.

Browns partner, Tim Hoeffgen, posted on Facebook, It is with great sadness that I announce that Timothy passed away surrounded by myself and friends, after a five-month battle with leukemia. Tim committed his lifes work to telling his story about his HIV cure and became an ambassador of hope.

The procedure itself is not routinely used to treat HIV because it is both too risky and aggressive. It is primarily used to treat certain types of cancer. In the case of both Brown and Castillejo, it was the combination of HIV and resultant cancers that are effectively treated with stem cell transplants, that made it feasible. Nonetheless, it gave patients hope that there may someday be a cure.

We owe Timothy and his doctor, Gero Hutter, a great deal of gratitude for opening the door for scientists to explore the concept that a cure for HIV is possible, stated Adeeba Kamarulzaman, president of the International Aids Society (IAS).

Sharon Lewin, director of the Doherty Institute in Melbourne, Australia, noted, Although the cases of Timothy and Adam are not a viable large-scale strategy for a cure, they do represent a critical moment in the search for an HIV cure. Timothy was a champion and advocate for keeping an HIV cure on the political and scientific agenda. It is the hope of the scientific community that one day we can honor his legacy with a safe, cost-effective and widely accessible strategy to achieve HIV remission and cure using gene editing or techniques that boost immune control.

Although largely a treatable disease, HIV/AIDS affects about 37 million people globally, and about 1 million people die from HIV-related causes each year. Treatment typically involves a cocktail of antiretroviral therapy, which HIV patients take their entire lives.

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Timothy Ray Brown, First Patient to Be Cured of HIV, Dies of Leukemia - BioSpace

Different Types of Childhood Cancer | INTEGRIS – Integris

If youre a parent, you know the thoughts, feelings and emotions that come with protecting your child. Youd do anything for them to ensure they live a long, healthy life. But what will you do when there are times when health conditions, such as childhood cancer, stand in the way?

Cancer in children is more common than you may think, as it accounts for the second-leading cause of death behind accidents. While a cancer diagnosis, or even the thought of your child developing cancer one day, is overwhelming, modern medicine has improved survival rates. As with any cancer, early detection is key. To help you familiarize yourself with childhood cancer, we asked Michael Confer, M.D., a radiation oncologist at the INTEGRIS Cancer Institute, about the different types of cancers, which signs to look for and how they can be treated.

Unlike adult cancers, which can result from the environment or exposure to certain things, childhood cancers result from genetic mutations that occur early on in life or before birth.

It all comes down to changes in genes. Your DNA contains information to make different types of cells in your body. In other words, your skin cells contain information to be brain cells, while your kidney cells contain information to be heart muscle cells. As cells mature, they become specialized, turning on and off certain genes to allow them to perform specific duties. Cells need to be able to replicate to replace damaged cells of the same category. They grow with help from genes called proto-oncogenes.

When your DNA changes, it leads to genetic mutations, and cells can become permanently activated. This can lead to cells duplicating uncontrollably, known as cancer.

Tumor suppressor genes slow down cell division. They repair DNA mistakes before cells divide and control cells internal death process (apoptosis or programmed cell death), Dr. Confer says. DNA mutations within tumor suppressor genes can also allow cells to duplicate uncontrollably. Children can be born with mutated proto-oncogenes or tumor suppressor genes in certain cells. These abnormally programmed cells lead to most childhood cancers.

So, what causes DNA changes? Your child can inherit genes from a parent that increases their risk of cancer or they can acquire these genes. Cancers from acquired, sporadic gene mutations are more common than those from inherited gene mutations 5% of all childhood cancers come from inherited mutations.

Breast cancer and ovarian cancer are the most common types of cancer caused by inherited DNA changes from BRCA1 or BRCA2 gene mutations. Even with how well-known these are, only 5 to 10% of breast cancer cases come from BRCA1/BRCA2 inherited mutations. Plus, breast cancer and ovarian cancer are more common in adults than children. Talk to your doctor or visit a genetic counselor if you have specific questions about inherited mutations.

Cancer can impact any part of your body, ranging from your bones and blood cells to your brain, spinal cord and other internal organs. You may be most familiar with leukemia, lymphoma, and brain and spinal tumors, since they are the most common. But, here is a full overview of cancers that commonly affect children, according to the American Cancer Society.

Leukemia: This is the most common type of cancer in children, accounting for 28% of cases. It generally starts in white blood cells and becomes fast growing (acute). Acute lymphocytic leukemia (ALL) and acute myeloid leukemia (AML) are the two most common types of leukemia. Three out of every four children with leukemia have ALL. This type of cancer starts in the lymphoid cells, called lymphocytes, whereas AML starts in myeloid cells. Chronic myeloid leukemia (CML) and chronic lymphocytic leukemia (CLL) are two types of rare cancers

Brain and spinal cord tumors: These types of cancers make up 26% of all cases. Brain tumors are more common than spinal cord tumors. The cancer generally starts in the lower part of the brain.

Neuroblastoma: This type of cancer affects infants and young children. Neuroblastoma starts in nerve cells during pregnancy and accounts for 6% of childhood cancers. Abdomen swelling is a common sign of neuroblastoma.

Wilms tumor: This type of cancer starts in the kidneys and impacts children who are 3 or 4 years old. It accounts for 5% of childhood cancers.

Lymphoma: Although lymphoma isnt as common as other types of cancers, youve likely heard of Hodgkin lymphoma and non-Hodgkin lymphoma, the two main types of cancer that show up in the lymphocytes. Non-Hodgkin lymphoma (accounts for 5% of childhood cancers) appears in younger children and is more common than Hodgkin lymphoma (accounts for 3%), which is more common in younger adults. You may notice a swollen lymph node under your childs arm or near their throat.

Rhabdomyosarcoma: This type of cancer develops in areas that your child uses to move their body, such as the head, pelvis, arms or legs. It accounts for 3% of childhood cancer cases.

Retinoblastoma: This cancer develops in the eyes, and your child is most at risk around the age of 2 until the age of 6. It accounts for 2% of childhood cancers.

Bone cancer: Bone cancer is more prominent in teens, but it still accounts for 3% of childhood cancers. There are two types, osteosarcoma and Ewing sarcoma, that show up via swelling around the bones. Ewing sarcoma is a less common type of cancer that is more prominent in older children and younger teens. Osteosarcoma, meanwhile, is more common in teens and in areas where bones grow quickly.

There isn't a one-size-fits-all guide to know if your child has cancer. In general, Dr. Confer says to keep an eye on any changes in your childs behavior, such as walking, eating, playing or sleeping. If theyre older, listen to any complaints they may have. Some cancers may produce a lump or swelling and pain in certain areas. Other symptoms include a loss of energy, weight loss, sudden eye or vision changes, frequent headaches with vomiting or a persistent fever that signifies the body is fighting an infection.

For example, leukemia, the most common type of childhood cancer, affects most children between the ages of 2 and 4. Typical symptoms include fever, bleeding, deep pain in the bones, small red spots on the skin called petechiae, bruises and enlarged lymph nodes.

Notify your childs pediatrician if any of these concerns arise. Aside from that, you should schedule your child for routine checkups and wellness visits.

Routine checkups and wellness visits help monitor normal growth and development. A good pediatrician-patient relationship helps the physician better identify subtle signs of cancer and gives parents a trusted sounding board for the concerns parents or children may have, Dr. Confer says.

Many childhood cancers have become increasingly treatable, leading to longer survival rates. Dr. Confer says acute lymphoblastic leukemia, lymphoma or kidney tumors known as Wilms tumor all have more than a 90% five-year survival rate (the percentage of patients who are alive five years after receiving treatment or a diagnosis).

In fact, the overall five-year survival rate for childhood cancers has improved from 58% in the mid-1970s to 84%, according to the American Cancer Society. But, certain types of aggressive cancer still exist. Diffuse intrinsic pontine glioma (DIPG), a rare brain tumor, is often cited as the childhood cancer with the poorest survival rate (less than 1% for five years).

No matter the diagnosis, continual hope and quality, proven therapies are the most important factors for children and families facing childhood cancers, Dr. Confer says.

Here are some of the most common forms of therapies to treat childhood cancer.

Surgery can help many patients, whether you need an entire tumor removed or a procedure to ease pain caused by a tumor. Your childs surgeon can also debulk a tumor, meaning they remove part of it and treat the rest with another method. Surgery has the highest success rate when its contained to one area, before the cancer has an opportunity to metastasize (spread to other parts of the body).

High doses of radiation help reduce cancer by either killing the cells or damaging their DNA to slow growth. Over time, these cells die and your body removes them. You can either receive internal or external radiation. External radiation comes from a beam that treats a specific body part, whereas internal radiation is in solid or liquid form. More specifically, brachytherapy is the medical term for solid internal radiation. Your doctor will place capsules, seeds or ribbons near the tumor. Systemic therapy is the medical term for liquid internal radiation. With this method, the radiation travels through your blood via a pill, injection or IV to kill cancer cells.

Chemotherapy comes in many methods of application, such as IV, oral, injection, topical or through a catheter, port or pump. Chemotherapy also kills healthy cells, which is one of the downsides. This is why many chemotherapy patients lose their hair and experience other side effects. Depending on the type and progression of the cancer, chemotherapy can help shrink a tumor to increase the success rate of surgery or radiation. Chemotherapy can also fight against any lingering cancer cells following surgery or radiation. Its also used to treat cancer that returns or metastasizes.

The immune system is your bodys way of defending itself against harmful germs, bacteria and viruses. When it comes to cancer, the immune system can have trouble recognizing and fighting off harmful cells because cancer starts in healthy cells. Immunotherapy helps your body pinpoint cancer kills to better defend against them. There are many types of immunotherapy treatments to boost your immune system. One type, chimeric antigen receptor (CAR) T-cell therapy, mixes your own T-cells with a virus that teaches the T-cells how to kill cancer cells.

Targeted therapy is a form of chemotherapy. But, as the name suggests, these drugs zero in on a specific area of the cancer cells. Depending on the drug, targeted therapy can change the protein levels in cancer cells or block chemical signals that help cancer cells grow. Other targeted therapy drugs can limit blood vessel production to cut off the cancer cells or distribute toxins to specifically kill the cancer while sparing healthy cells.

Stem cells, which originate in the bone marrow, make red blood cells, white blood cells and platelets. Leukemia and lymphoma start in the blood cells, causing damage to the cells your body needs to function. A stem cell transplant involves destroying cancer cells via chemotherapy and/or radiation before replacing them with new, healthy cells. This allows doctors to use stronger doses of chemotherapy or radiation knowing new cells, via a transplant, will replace old, damaged cells. Stem cell transplants can come from your own cells or the cells of another person. Donated cells are often more effective since they can help kill off cancer cells.

While you cant do anything to prevent your child from developing cancer, you can be proactive by scheduling regular checkups and looking out for warning signs and symptoms. Contact an INTEGRIS pediatrician if you have any concerns, and they can refer you to an oncologist at the INTEGRIS Cancer Institute.

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Different Types of Childhood Cancer | INTEGRIS - Integris

Gene therapy company Taysha completes sprint from first funding to IPO – BioPharma Dive

Dive Brief:

Gene therapies have graduated from the laboratory bench to doctor's offices. The first wave of agents, Roche's Luxturna and Novartis' Zolgensma, are already altering the course of disease in two conditions, respectively an inherited form of blindness and the degenerative, often fatal disease spinal muscular atrophy.

In both cases those treatments were developed by companies that had relatively smaller pipelines, Spark Therapeutics for Luxturna and AveXis for Zolgensma.

Enter Taysha, which is headed by a former AveXis business development vice president, R.A. Session II. The company has highly ambitious hopes to launch a new product every two to three years, with the goal of building a durable business around adapting its technology across many diseases driven by defects in single genes.

The company also plans to build a commercial-scale manufacturing plant from the start, aiming to avoid some of the setbacks that can occur when production moves from facilities built to supply clinical trials.

The linchpin of the company's business is an agreement with UT-Southwestern, under which Taysha funds research and can obtain exclusive rights to experimental therapies for central nervous system disorders, through the end of 2021. Neurodegenerative disorders have proven challenging for some gene therapies because of the difficulties in delivering the viral vectors that carry gene replacements to brain tissue.

In spinning out Taysha, UT-Southwestern took an ownership stake in Taysha, amounting to 2.2 million shares, which is now worth more than $40 million. These ownership stakes have become more common with gene therapies in particular, as big pharma companies have been reluctant to license intellectual property straight out of university laboratories.

Taysha's lead project is called TSHA-101, which seeks to treat a condition called GM2 gangliosidosis, a disorder in which lipid accumulation destroys nerves in the brain and spinal cord. The first clinical trial is scheduled to begin in Canada by the end of 2020.

The company's shares rose following their first trades on the NASDAQ exchange, gaining 20% to close the day at $24.06.

Editor's note: This story was updated to reflect the share price at the close of trading.

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Gene therapy company Taysha completes sprint from first funding to IPO - BioPharma Dive

Focused on the future: Innovative college programs in N.J. pave way for the real world – Jersey’s Best

New Jersey students browsing a course catalog will find many programs of study thatwerentthere when their parents went to college.

Some programs seem to be exactly appropriate for the time we are in.

Theres cyberpsychology at New Jersey Institute of Technology (NJIT), environmental studies at Rutgers, financial technology at Seton Hall and cannabis studies at Stockton University.

As perhaps the largest mass demonstrations in American history have drawn attention to prejudice against minorities, including police brutality and inequity in the impact and treatment of the COVID-19 pandemic, The College of New Jersey (TCNJ) offers a minor in social justice.

Its so interesting that it fits the moment, but it is not a new idea for us, said Dr. PiperKendrixWilliams, a professor in the English and African American Studies departments at TCNJ.

The idea for the minor is three or four years old, and we worked on it for about two years, she said.

New Jersey tends to have segregated areas white suburbs and minorities in the cities, she said. Even in college, students experience can be really segregated.

Our students need an education in social justice, she said. Every student in the college needs some understanding of race, gender and inclusion. I can see a lot of students wanting this.

Williams said the program is a natural outgrowth of TCNJs Bonner Scholars program in which participants are required to do 300 hours of community service in the Greater Trenton area.

The minor requires five courses: Introduction to Social Justice, Introduction to Womens and Gender Studies, Introduction to the Sociology of Race and two junior-level electives.

Activism cant come until youre educated, until you know what youre fighting for, Williams said. Higher education has to step up to what is relevant in the world.

Dr. JulieAnciswill be the director of NJITs new program in cyberpsychology, offering a Bachelor of Science degree. It is the first such major in New Jersey and NJITs first foray into the behavioral sciences.

Im very excited about this program because its truly innovative, saidAncis, who has been a professor of psychology and associate vice president of diversity at Georgia Tech.

It is designed for students with an interest in psychology and human behavior, she said, noting that projections call for at least an 11% job growth in the field in the next 10 years.

The program will use existing research and literature to prepare students to solve real-world problems and apply their skills to a variety of domains,Ancissaid.

Potential career fields will include cyber security, games We want games to be a force for good, she said telepsychology, virtual reality, app development, digital health and any positions related to online engagement.

We will be exploring all aspects of technologically interconnected human behavior through the lens of psychology,Ancissaid. We will look at ways to enrich online experiences and decrease anxiety, explore the psychology of cyber threats both for perpetrators and victims factors that influence privacy precautions such as perception of risks, cyberbullying, ethics and many other areas.

The ways in which we learn, socialize and communicate have been transformed, she added. Were in a totally new worldapproximately 60% of the worlds population is using the internet.

Anciswill teach Foundations of Cyberpsychology, which has a waiting list for registration.

I expect this really to grow, she said.

NJIT also is offering a new undergraduate major in forensic science, and certificates in big data, data mining, and cell and gene therapy.

Cannabis has been on college campuses for decades, but with medical marijuana now legal in 33 states and recreational marijuana legal in 11, a new industry has spawned.

The university was interested in having a scientist involved because there might be perceptions that illegal activity was involved, said Dr. EkaterinaSedia, a plant biologist and coordinator of the cannabis studies minor at Stockton University. The program is very much based in science.

OK, we have a new industry, its coming, its here and theyre going to be hiring. We want to get those jobs.

About 50 students are enrolled in the program, which started a year ago.

I anticipated the demand,Sediasaid. I was a little surprised how quickly it became known. We have students from a variety of majors, including business, environment and sustainability, and communications.

The program also looks at hemp and CBD products, and includes such aspects as banking, cybersecurityand regulatory compliance. It studies the non-psychoactive aspects of cannabis, such as use in cosmetics, too.

Its not necessary that youd be growing it yourself, but you may be involved with a business that grows it,Sediasaid. I have a student who says she wants to be the Johnson & Johnson of cannabis.

Elven Riley of Seton Halls Stillman School of Business saidthe financial technology major FinTech for short is aimed at preparing students for jobs where finance and the digital world intersect.

In the finance department we see that banking and brokerages and, to some extent, insurance are reducing their traditional workforces and moving to more technology savvy employees, he said. There are just thousands of jobs out there. New York and London are hotbeds.

The major is designed for business students to become literate in the language of technology.

We think it will put our students at the head of the queue to get those jobs, said Riley, who had a 30-year career in investment banking. Were getting attention from the industry that they want these students.

Seton Hall now has students who pursue dual majors in finance and information technology. Forty to 50 students are expected to switch to the FinTech major.

This is a mash-up of the two concentrations, Riley said. Looking around higher education, thereare not a lot of these designed programs.Theresnot a lot of faculty in higher education that has experience in this space.

Theres big interest, lots of jobs, very exciting, but also very challenging, he said.

The possible careers are in big data, algorithmictradingand edge computing.

Theres no part of the financial industry that doesnt involve technology, Riley said. You need people who are trained and literate inboth of these.

We do think incoming students will seek this out. We think this will attract high-quality students to our program. We would be remiss not to go in this direction.

Rutgers University-New Brunswicks School of Arts and Sciences has launched a new major in environmental studies that brings together 17 science and humanities programs.

This appeals to up-and-coming generations who have grown up with the environment being at the forefront of their world, said Dr. Paul OKeefe, whose specialty is geography.

Different in focus from environmentalscience,the environmental studies major will prepare liberal arts students to be professionals on environmental issues, OKeefe said.

We currently have 26 students enrolled in the environmental studies minor and expect several to transition to the major along with new entrants to the program, he said.

Students in all majors must take Environmental Techniques courses, which give them skills applicable to many workplaces. There also is an optional internship component for students.

Rutgers also offers a minor in agroecology in the School of Environmental and Biological Sciences in New Brunswick. It appeals to a small number of students from a wide variety of majors.

I have had landscape architects and nutrition majors who are interested in how we grow food and food systems, said Dr. Mark Robson, Board of Governors Distinguished Service Professor in the Department of Plant Biology. I have had kids from the business school who want to know about the costs of growing food. I have had kids in our traditional ag(riculture)and food systems major that used to be ag(riculture) science, and then I have students who are just interested in ecology in general and this caught their eye.

So, given its general nature and the fact that we all eat, it seems to interest a lot of students, he said.

Students have gone on to work for state environmental agencies and the U.S. Department of Agriculture, and some have gone into farming as well, Robson said.

Other new programs at Rutgers include majors in cinema studies, Japanese and Korean, and minors in African languages, archaeology, Holocaust studies, and medical ethics and health policy.

Kean University has started a new major in exercise science in its School of Health and Human Performance. It can lead to careers in physical and occupational therapy, chiropractic, cardiac rehabilitation, and sports medicine, and lead to work in the fitness industry.

Some of our students become entrepreneurs and start their own fitness businesses, said Dr. Adam Eckart, program coordinator, who has worked as a full-time trainer and whose bachelors and masters degrees are from Kean.

It can be a feeder for graduate health programs, including medical school, he said.

Eckart said there are 215 exercise science majors, and the program is expected to grow by 40 to 50 students a year.

Health care organizations will be looking in the future to refer patients to exercise professionals, he said. Students need an insight into where the field is headed.

He said most of health care spending results from chronic conditions that can be avoided with intervention through diet and exercise, pointing to the motto of the American College of Sports Medicine: Exercise is medicine.

Seton Hall also has launched a dual major in religion and law, which will allow students to graduate in six years with a bachelors degree in religion and a juris doctor in law.

Were very excited about it, said Dr. Brian Sheppard of Seton Hall Law School in Newark, who said the religion major requires a rigorous study of texts, which overlaps with law school rigor. He said it appeals to students who are interested in working in the nonprofit sector.

We like the idea of building this bridge, Sheppard said. We get some incredible students from Seton Hall undergraduate. This just strengthens that.

It is one ofseveralthree-plus-three programs offered by Seton Hall, leading to both undergraduate and graduate degrees in six years.

You use your first year of studies in law school as your fourth year of college, Sheppard said. It really offers a premium to students who want both degrees. It allows you graduate with less debt. Debt can be a real obstacle to careers in social justice.

And Seton Halls School of Diplomacy and International Relations is offering non-majors a certificate in global affairs.

Acting Dean Courtney Smith said many diplomacy majors minor in languages and other disciplines in Seton Halls other schools.

This is an opportunity for us to offer a reciprocal service, he said, to better serve students already here.

The certificate will require four classes that will give students a better grasp of global affairs, Smith said, since many of the issues facing society in the future require an international approach, including climate change.

He said students can take a broad or narrow approach in choosing their classes. It requires only 12 credits, rather than the 18 required for a minor.

Kean also has started a new dual admission program Pathway to Kean that offers students whodontinitially meet university admissions standards a path toward earning a bachelors degree.

Qualified students are given conditional acceptance to the universityas long asthey complete their associate degree at one of Keans partner county colleges. The university will provide counseling and support services while the students are attending county colleges.

The program begins this fall in partnership with Essex County College, Middlesex County College, the County College of Morris, Ocean County College, Union County College and Warren County Community College. It is expected to include more New Jersey county colleges over the next two years.

Students in the program will have their applications sent to their respective county colleges. Kean will work with each county college admissions office to ease their enrollment into the program.

Kean will provide support services on the county college campuses and remotely for students in the program.

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Focused on the future: Innovative college programs in N.J. pave way for the real world - Jersey's Best

Burosumab Is a ‘Game Changer,’ Effective in All Subgroups of XLH – Medscape

A recently approved agent, burosumab (Crysvita), was better than placebo across a range of efficacy outcomes for 14 predefined subgroups of adults with X-linked hypophosphatemia (XLH), new research shows.

The authors analyzed data from the initial 24-week randomized blinded phase of the pivotal phase 3 trial that led to regulatory approval of this drug in the United States in 2018 for XLH, a rare form of rickets characterized by low serum phosphorus levels, skeletal defects, pain, and stiffness.

As in the main analysis, in the subgroups, among patients who received burosumab, serum phosphorus levels were improved, and outcomes were better on the following measures: Western Ontario and McMaster Universities Arthritis Index (WOMAC) stiffness scale, the WOMAC physical function measure, and the Brief Pain Inventory (BPI), which were the main efficacy outcomes. Improvements were seen for many other outcomes as well.

Maria-Luisa Brandi, MD, Careggi University Hospital, Florence, Italy, presented the new subanalysis during the virtual American Society of Bone and Mineral Research (ASBMR) 2020 annual meeting.

The subgroup results were consistent with the overall trial findings, "showing a favorable direction of effect of burosumab relative to placebo" except for results in patients recruited in Asia and non-White patients; those results were considered inconclusive because there were too few participants in those categories, she told Medscape Medical News,.

Lorenz Hofbauer, MD, scientific chair of the ASBMR meeting, said that the take-away message is that the drug "works to reduce pain and disability" in adults with XLH with more severe/less severe symptoms, and "it provides new hope for many patients suffering from this disease," he told Medscape Medical News.

Burosemab also appears superior to what has previously been considered standard therapy for XLH, phosphate/calcitriol, the experts say.

"The disease prevalence is 1 to 9 in a million," Brandi said. "Undiagnosed adults are treated by the doctor that makes the diagnosis, usually a nephrologist or a rheumatologist or a bone doctor; this depends on the prevalent complications in a given patient. The endocrinologist who treats this patient is the one expert in bone disorders."

Hofbauer noted, however, that "[r]are is relative. If you run a bone clinic, you will see four to five patients with XLH; if you are a regional center, 20 to 30 patients. People with rare disease travel more than 1000 miles to see experts."

The US Food and Drug Administration approved burosumab for use in children and adults with XLH 2 years ago. The European Medicines Agency (EMA) approved it for use in children.

The drug is expected to be approved by the EMA for adults with XLH some time this year, said Hofbauer, who is from Dresden Technical University, Dresden, Germany.

Burosumab is a "game changer" with respect to previous treatments, he stressed.

This study is one of the top five clinical abstracts of the ASBMR meeting, which are selected on the basis of "scientific content/novelty, making a difference in clinical practice," Hofbauer explained. He noted that "new drugs that work are always in the top ranks."

Craig Munns, PhD, who was senior author of a recent review about burosumab, agrees.

"Burosumab is transformative, as it is a paradigm shift in the way we manage XLH," he told Medscape Medical News.

"Standard therapy for children is with oral phosphate and calcitriol, and many adults do not receive any therapy," said Munns, from the University of Sydney, Sydney, Australia.

"Phosphate and calcitriol need to be taken multiple times per day, is an incomplete therapy, and has many complications. Burosumab offers a 2-weekly (children) or 4-weekly (adult) dosing regime with superior outcomes compared to no treatment or phosphate/calcitriol," he emphasized.

"Burosumab is an anti-FGF-23 [antifibroblast growth factor-23] antibody for a rare genetic disease, XLH, in which the gene for PHEX is defective," Hofbauer explained.

"PHEX is an enzyme that clears FGF-23; if it does not work, then FGF-23 accumulates in the body and causes phosphate wasting with wide consequences for bone, muscle, and joints. Burosumab is a smart approach, since it blocks these excessive FGF-23 effects."

Children with XLH have rickets, deformities in the lower skeleton, and short stature, Brandi noted, whereas adults have fractures, pseudofractures, enthesopathy (calcification of joint capsule, tendon insertions, and ligaments), pain, stiffness, and impaired physical function.

However, "treatment with oral phosphate and vitamin D is associated with nephrocalcinosis and hyperparathyroidism," she said.

In the phase 3 trial, 134 adults (aged 18 to 65 years) with XLH were randomly assigned in a double-blind manner to receive either burosumab or placebo for 24 weeks, followed by 24 weeks of open-label burosumab. The patients' serum phosphorus levels were <2.5 mg/dL, and they were experiencing measurable bone/joint pain.

Baseline characteristics were similar for the patients who received placebo (66) and those who received burosumab (68). The mean age of the patients was 40 years; 65% were women; and 81% were White.

The current exploratory analysis examined efficacy outcomes in patients grouped according to the following factors and characteristics: sex; age (41 years or >41 years); race (non-White, White); region (Asia, North America/Europe); baseline WOMAC pain score; WOMAC total pain; WOMAC stiffness; WOMAC physical function; BPI worst pain; BPI average pain; opioid use; pain medication use; active fractures and pseudofractures; and 6-minute walking test distance.

The efficacy outcomes were as follows: serum phosphorus level (primary outcome), BPI worst pain, WOMAC stiffness, and WOMAC physical function (key secondary outcomes); and WOMAC pain, WOMAC total score, BPI average pain, BPI pain interference, BPI worst fatigue, BPI global score, patient global impression (PGI), and 6-minute walking distance.

In the overall cohort, at 24 weeks, in comparison with patients who received placebo, patients who received burosumab had favorable responses with respect to serum phosphorus level, WOMAC stiffness (P =. 012),WOMAC physical function (P = .048), and BPI worst pain (P = .092, not significant), as well as significant improvements in WOMAC total score and the 6-minute walk test. There were nonsignificant improvements in WOMAC pain and BPI average pain.

In the subgroup analysis, burosumab was superior to placebo for the primary outcome (serum phosphorus) in all subgroups. It was also superior to placebo for the key secondary outcomes (worst pain, stiffness, and physical function) across all subgroups except for patients from Asia (18 patients) and non-White patients (26).

The study was funded by Kyowa Kirin in partnership with Ultragenyx. Brandi receives consultancy and speaker fees as well as research grants from Kyowa Kirin and other pharmaceutical companies. Munns has received research funding from Kyowa Kirin.

The American Society of Bone and Mineral Research (ASBMR) 2020 Annual Meeting Virtual Event: Presented September 12, 2020.

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Burosumab Is a 'Game Changer,' Effective in All Subgroups of XLH - Medscape

10 Forgotten Spiderman Sidekicks They Need To Bring Back | CBR – CBR – Comic Book Resources

Spider-Man might not be a superhero typically known for his sidekicks, but he's definitely had some over the years. And these deserve a comeback.

It took Spider-Man a long time to finally relent and join a superhero team. Even then, it took the combined effort of Captain America and Iron Man to convince him. Before this, he had only been in the Avengers for a paltry four issues and had flirtations with the Defenders and the Fantastic Four. Even his Avengers membership had been as a reserve member.

RELATED: The Top Ten Spiderman And Deadpool Team Ups, Ranked

That does not mean the wall-crawler was exclusively a solo act. In fact, the long-running Marvel Team-Up featured Spider-Man in almost every issue of its thirteen-year run. Spider-Man was not averse to working with others, even if he did annoy most of his partners with his awkward gags. In fact, Spider-Man has nurtured and partnered with a lot of othercharacters in his time. From teenage runaways to reformed criminals, the web-slinger has had his fair share of sidekicks.

Robert Farrell has turned up on both sides of the law in his brief appearances. As well as being an adversary and sidekick of Spider-Man, he has also assisted the Avengers and worked undercover for S.H.I.E.L.D. As a child, realizing that the odds were stacked against him as a poor, black youth from the slums, he developed a rocket-powered skateboard. He used this to commit minor robberies to pay rent and bills for his mother.

After a stint on Ryker's Island, he reformed and began to assist Spider-Man. His cool, eighties look is very current. With his background that touches on themes inherent in the BLM movement, the world needs a new Rocket Racer story.

In her short stint in comics, Jackpot has developed quite a backstory. She began as an employee at Oscorp and was exposed to experimental gene therapy. This put her in a coma for four months, after which she awoke, gave birth, and realized she had developed super strength.

She was only a superhero briefly beforequitting to raise her daughter. In a very modern twist, she then sold her identity and brand. The new Jackpot teamed up with Spider-Man until her death when the outfit returned to its original occupant. The single mother superhero narrative has been approached in comics recently, but fans would be impressed to see a story involving a working superhero motherwho doesn't have a big team behind her.

Cloak and Dagger are two of the most recognizable of Spider-Man's sidekicks. They are so iconic, they even got their own television series. Since their debut in 1982, Tyrone and Tandy have been members of the Runaways, X-Men, and also fought alongside Captain America in the Civil War. They have even fought against heavyweights such as Doctor Doom and the Beyonder.

Fans adore Cloak and Dagger most when they are fighting street-level villains alongside Spider-Man. They were last seen guarding Hong Kong against the influence of Mr. Negative. Fans eagerly await their return home to New York.

Puma is a very complex ally to Spider-Man, and deserves to be more of a partner to Spidey than a sidekick. He is of Native American descent, and fiercely loyal to his heritage. Though a man of morals and good standing, he has also clashed with the web-slinger on some occasions.

RELATED: 5 Spiderman Villain Who Are Actually Sympathetic (& 5 Who Are Pure Evil)

His powers are a mix of martial arts training, technology, and Native American mysticism. As a successful businessman, it is surprising that his company, Fireheart Enterprises,never made deals with Parker industries. In a world polarized between the values of tradition and technology, fans would delight in a renewed version of the Puma.

Silver Sable has everything. She is a businesswoman, bounty hunter, a princess, and the owner of an international company. Despite lacking any actual super powers, she has access to a wealth of technology and is a fierce martial artist.

While not forgotten, Silver Sable is longoverdue a permanent comeback in the world of the wall-crawler. Her antagonistic nature and single-mindedness could either be a blessing or curse for Spider-Man. The idea of her as a private security consultant, as explored in the recent Spider-Man videogame, could see her tip the wall crawlers world upside down.

Cardiac's last appearance was during the Superior Spider-Man era when Doctor Octopus was inhabiting the body of Peter Parker. Despite friction, they decided they would still work together on medical research projects in the future. In fact, Cardiac even made Otto Octavius feel guilty about his past schemes, which is just the kind of character Cardiac is.

Elias Wirtham is a physician and owner of a biomedical research company who became Cardiac to right the wrongs ofgreedy pharmaceutical companies after the death of his brother. He is a medical Robin Hood, robbing drugs and equipment for those in need. While this can get him into trouble, his intent is always noble and he has partnered Spider-Man on many occasions.

Though he partnered with Spider-Man during the '90s, Nightwatch was anything but a good guy. In fact, in a bid to erase his former existence from history, he condemned a whole town to genocide. His motivation to be a hero was founded on the premise that they had the best retirement plans.

Nightwatch was last seen in the pages of She-Hulk, who brought him to justice using the law. Now atoning for his crimes in jail, a return from Nightwatch would finally allow him to make amends and reconcile his past sins. Who would be better equipped to help him do that than Spider-Man?

Of all the sidekicks that Spider-Man has had in his tenure, the most memorable come from a kooky TV series. Spider-Man and his Amazing Friends might not make much sense in terms of Spidey's comic book canon, but in the early '80s, this team-up cartoon was huge.

RELATED: Spider-Man: Ten New Characters Introduced During The Clone Saga

The team formed in episode one, movinginto Aunt May's apartment with a small dog and deciding to fight crime. For a modern update, the backstory may need reworking. They did have a brief reunion inanIceman mini-series.There's no doubt that fans would relish the chance to read a retro Amazing Friends comic in the vein of X-Men '92.

The White Tiger belongs to a legacy and has been a moniker adopted by five different heroes in the history of Marvel. The first White Tiger, Hector Ayala, was the hero most affiliated with Spider-Man. Hector carried a jade amulet that would let him transform into a White Tiger. He teamed up with Spider-Man many times in the'70s until he was accused of a crime he did not commit.

Despite the best efforts of his lawyer, Matt Murdock (Daredevil), he was found guilty and later assassinated. Ava Ayala, his younger sister, was the most recent to take uphis mantle of White Tiger. Whether she still has the White Tiger amulet or another has taken up the moniker remains to be seen.

Solo is a work for hire mercenary, similar in his outlook and abilities to The Punisher. He will use lethal means to get what he wants. Though this often brings him into conflict with the web-slinger, it has also meant that they have worked side by side on many occasions.

Solo's adventures have since taken him to some wild and wonderful situations. He has worked for Deadpoolas one of the "mercs for money"and even with S.H.I.E.L.D. in the past, though he is always best when trying to tow the line alongside Spider-Man.

NEXT: Spider-Man: The 10 Worst Things Black Cat Has Ever Done

Next Iron Fist Vs. Elektra: Who Would Win?

Carl Jackson is a typical English gentleman currently residing in Eastern Europe. He lives with his two cats, wife, and Neo Geo arcade machine. He is an avid retro gamer, collector of the eighties toy line M.A.S.K. and enjoys getting new ink on his Marvel vs Capcom sleeve.

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Genentech Presents New Data From Multiple Phase III Studies of Tecentriq in Triple-Negative Breast Cancer at ESMO Virtual Congress 2020 – Business…

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that it presented the latest results from three Phase III studies from the Tecentriq (atezolizumab) clinical development program in triple-negative breast cancer (TNBC) at the European Society for Medical Oncology (ESMO) Virtual Congress 2020.

While we have made great progress in the treatment of many forms of breast cancer, TNBC remains an aggressive and difficult-to-treat disease, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. We are proud of our work to address challenges and advance scientific understanding of cancer immunotherapy in the context of distinct chemotherapy regimens and in various TNBC treatment settings. Although the IMpassion131 study did not reach its endpoint, we are pleased to bring new treatment options for some TNBC patients, and remain committed to improving the lives of all women with early and advanced stages of this disease.

Results from the Phase III IMpassion031 study, evaluating Tecentriq in combination with chemotherapy (Abraxane [albumin-bound paclitaxel; nab-paclitaxel]; followed by doxorubicin and cyclophosphamide) in comparison to placebo plus chemotherapy (including nab-paclitaxel) demonstrated a statistically significant and clinically meaningful improvement in pathological complete response (pCR) for the treatment of people with early TNBC, regardless of PD-L1 expression. pCR was observed in 57.6% (95% CI: 49.7-65.2) of patients treated with Tecentriq in combination with chemotherapy, an increase of 16.5% from 41.1% (95% CI: 33.6-48.9) in patients treated with placebo plus chemotherapy (one-sided p=0.0044, significance boundary = 0.0184) in the intention-to-treat (ITT) population. The safety profile was consistent with the established profile of the individual medicines and no new safety concerns were identified.

The IMpassion031 study is the second positive Phase III study from Genentech demonstrating the benefit of Tecentriq in TNBC, and the first Tecentriq study to demonstrate benefit in early TNBC. Tecentriq in combination with nab-paclitaxel is currently approved in more than 70 countries worldwide, including the United States and across Europe, for the treatment of adults with unresectable locally advanced or metastatic TNBC in people whose tumors express PD-L1 (IC1%).

The final overall survival (OS) analysis of the Phase III IMpassion130 study, evaluating Tecentriq in combination with nab-paclitaxel compared with placebo plus nab-paclitaxel as a first-line treatment for patients with metastatic TNBC, was consistent with the first and second interim analyses. There was no significant difference in OS between the treatment groups in the ITT population. Clinically meaningful improvements in OS were seen with Tecentriq plus nab-paclitaxel in PD-L1-positive patients. The magnitude of OS improvements with Tecentriq in PD-L1-positive patients remained clinically meaningful, with an increase of 7.5 months in median OS with Tecentriq plus nab-paclitaxel compared with placebo plus nab-paclitaxel (hazard ratio [HR]=0.67 [95% CI: 0.530.86]). However, this result could not be formally tested due to the prespecified statistical testing hierarchy. The cumulative safety of the Tecentriq plus nab-paclitaxel combination remains consistent with the previously reported safety data for this study and the known risks of individual study medicines. No new safety concerns were identified with longer follow-up.

Finally, results from the Phase III IMpassion131 study, evaluating Tecentriq in combination with paclitaxel compared with placebo plus paclitaxel as a first-line treatment for patients with metastatic TNBC, did not show significant improvement for progression-free survival (PFS) in the PD-L1-positive population (HR=0.82 [95% CI: 0.60-1.12]). The OS data showed a negative trend; however, the study was not powered for the secondary endpoint of OS, and OS data were immature at the time of analysis (initial HR=1.55 [95% CI: 0.86-2.80] in the PD-L1-positive population, based on 21% of patients with an event; updated HR=1.12 [95% CI: 0.76-1.65], updated analysis based on 41% of patients with an event). The safety profile of Tecentriq plus paclitaxel was consistent with the established safety profile of the individual study medicines and no new safety concerns were identified.

Genentech has an extensive development program for Tecentriq, including multiple ongoing and planned Phase III studies across several types of lung, genitourinary, skin, breast, gastrointestinal, gynecological, and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMpassion031 study

The IMpassion031 study is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane [albumin-bound paclitaxel; nab-paclitaxel]; followed by doxorubicin and cyclophosphamide) in comparison to placebo plus chemotherapy, in people with previously untreated, early TNBC. The primary endpoint is pCR using the American Joint Committee on Cancer (AJCC) staging system in the intention-to-treat (ITT) population and in the PD-L1-positive population. Secondary endpoints include overall survival (OS), event-free survival, disease-free survival and quality of life measures.

About the IMpassion130 study

The IMpassion130 study is a Phase III, multicenter, randomized, double-blind study evaluating the efficacy, safety and pharmacokinetics of Tecentriq plus nab-paclitaxel compared with placebo plus nab-paclitaxel in people with unresectable locally advanced or metastatic TNBC who have not received prior systemic therapy for metastatic breast cancer. The study enrolled 902 people who were randomized equally (1:1). The co-primary endpoints are PFS per investigator assessment (RECIST 1.1), and OS in the ITT population and PD-L1-positive population. Secondary endpoints include objective response rate and duration of response.

About the IMpassion131 study

The IMpassion131 study is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of Tecentriq in combination with paclitaxel, in comparison to placebo plus paclitaxel, in people with previously untreated, inoperable, locally advanced or metastatic TNBC. The study enrolled 651 people who were randomized in a 2:1 ratio to receive Tecentriq or placebo plus paclitaxel. The primary endpoint is PFS per investigator assessment (RECIST 1.1) in the PD-L1-positive population, followed by intention-to-treat (ITT) population. Secondary endpoints include OS, objective response rate, and duration of response in the PD-L1-positive and ITT populations.

About triple-negative breast cancer

Breast cancer is the most common cancer among women worldwide. According to the American Cancer Society, close to 280,000 people in the United States will be diagnosed with invasive breast cancer, and more than 42,000 will die from the disease in 2020. Breast cancer is not one, but many diseases based on the biology of each tumor. In triple-negative breast cancer, tumor cells lack hormone receptors and do not have excess HER2 protein. Approximately 15 percent of breast cancers are triple-negative based on the results of diagnostic tests. It is an aggressive form of the disease with few treatment options.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Tecentriq U.S. Indications (pronounced t-SEN-trik)

Tecentriq is a prescription medicine used to treat adults with:

A type of bladder and urinary tract cancer called urothelial carcinoma.

Tecentriq may be used in patients with urothelial carcinoma if their bladder cancer has spread or cannot be removed by surgery, and if they have any one of the following conditions:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called non-small cell lung cancer (NSCLC).

Tecentriq may be used alone as the first treatment in patients with lung cancer if:

Tecentriq may be used with the medicines bevacizumab, paclitaxel, and carboplatin as the first treatment in patients with lung cancer if:

Tecentriq may be used with the medicines paclitaxel protein-bound and carboplatin as the first treatment in patients with lung cancer if:

Tecentriq may be used alone in patients with lung cancer if:

A type of breast cancer called triple-negative breast cancer (TNBC).

Tecentriq may be used with the medicine paclitaxel protein-bound in patients with TNBC when their breast cancer:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called small cell lung cancer (SCLC).

A type of liver cancer called hepatocellular carcinoma (HCC).

Tecentriq may be used with the medicine bevacizumab when a patients liver cancer:

A type of skin cancer called melanoma.

Tecentriq may be used with the medicines cobimetinib and vemurafenib when a patients melanoma:

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

The most important information about Tecentriq is:

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life-threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

The most common side effects of Tecentriq when used in TNBC with paclitaxel protein-bound include:

The most common side effects of Tecentriq when used in hepatocellular carcinoma with bevacizumab include:

The most common side effects of Tecentriq when used in melanoma with cobimetinib and vemurafenib include:

Tecentriq may cause fertility problems in females, which may affect their ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects of Tecentriq.

Report side effects to the FDA at (800) FDA-1088 or Report side effects to Genentech at (888) 835-2555.

Please visit for the full Tecentriq Prescribing Information for additional Important Safety Information.

About Genentech in cancer immunotherapy

Genentech has been developing medicines to redefine treatment in oncology for more than 35 years, and today, realizing the full potential of cancer immunotherapy is a major area of focus. With more than 20 immunotherapy molecules in development, Genentech is investigating the potential benefits of immunotherapy alone, and in combination with various chemotherapies, targeted therapies and other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system.

In addition to Genentechs approved PD-L1 checkpoint inhibitor, the companys broad cancer immunotherapy pipeline includes other checkpoint inhibitors, individualized neoantigen therapies and T cell bispecific antibodies. For more information visit

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit

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Genentech Presents New Data From Multiple Phase III Studies of Tecentriq in Triple-Negative Breast Cancer at ESMO Virtual Congress 2020 - Business...

BCS Announces Partnership With 2nd.MD to Improve Member Healthcare Cost, Quality, and Outcomes – PRNewswire

CHICAGO, Sept. 16, 2020 /PRNewswire/ -- BCS Financial (BCS) announces today it has partnered with 2nd.MD to provide top-tier medical navigation, expert consultation, and personalized second opinions to four million policyholders. Under the agreement, members covered under BCS Stop Loss and Excess of Loss policies can work with highly skilled, experienced nurses to help them navigate diagnoses, virtually connect with elite doctors who specialize in their specific condition, and receive assistance finding a local doctor or specialist in their area at no cost to them.

As the number one reinsurer of Blue Cross Blue Shield plans, BCS offers a wide range of products and services to help insurance carriers and their members navigate healthcare costs, particularly those exceeding $500,000 annually. The partnership is one of several features included in BCS Financial's Risk Navigator solutions, a highly predictive and actionable approach to managing claims risk.

"Second opinions can play a vital part in ensuring healthcare costs are not wasted on unnecessary or incorrect care," said Peter Costello, presidentand chief executive officer, BCS Financial. "BCS is committed to helping our policyholders and their members manage their risks while improving healthcare outcomes."

Members see direct benefit from the second-opinion consultation. "Members facing a complex diagnosis, surgeryor healthcare decision often find it difficult to navigate the healthcare system and manage costs," said Chris Kurtenbach, vice president, Services & Operations, BCS Financial. "With this partnership, we can help members get the appropriate care they need by connecting them with board-certified, elite specialists from top medical institutions, and reducing their costs along the way."

In many cases, the second opinion can change the treatment trajectory 85% of 2nd.MD's consultations result in improved treatment plans, 35% lead to alternative diagnoses, and nearly 30% of surgery consults result in surgery cancellation.

This is an especially important service as the market for gene therapy and cellular immunotherapy continues to ramp up. An estimated 50-75 therapies will be approved for the U.S. market by 2030, with costs for cellular immunotherapies averaging around $400,000, and gene therapy costs estimated in the $2-$4 million range. For those with complex cancers, rare or orphan conditions, and aggressive recommendations, access to top specialists can make all the difference.

"Better care and outcomes for members is always our priority," said Costello. "This partnership is just one of the ways we are delivering value to our Blue Cross and Blue Shield partners and their members by helping them continue to manage healthcare costs and quality."

This service is currently available to members covered by BCS Stop Loss and Excess of Loss policies.

About BCS Financial Corporation (BCS)

BCS Financial Corporation has more than 70 years of experience delivering a wide-range of insurance and financial solutions for Blue Cross and Blue Shield organizations nationwide. Through its subsidiaries, BCS Insurance Company and 4 Ever Life Insurance Company, BCS is licensed in all 50 states and is rated A- (Excellent) by A.M. Best. BCS Financial is headquartered in Oakbrook Terrace, Illinois. Learn more at

About 2nd.MD

2nd.MD offers medical certainty by connecting you with board-certified, leading specialists from across the country for an expert second opinion via video or phone within three to five days, from the comfort of your own home. From the minute you reach out, 2nd.MD's Care Team of nurses will put you at the center of your medical care by coordinating all the details. This includes understanding your health goals, gathering all of your medical records, and connecting you with a doctor who specializes in your specific medical condition, so that you can focus on what matters most getting the best care possible. No travel. No hassle. No cost. If you haven't used 2nd.MD before, it can be life-changing. In fact, 98% of people who use 2nd.MD recommend it to their friends and family.

For more information, please contact Nathan Post at 630-472-7860.

SOURCE BCS Financial Corporation

BCS Announces Partnership With 2nd.MD to Improve Member Healthcare Cost, Quality, and Outcomes - PRNewswire

Novus Therapeutics Announces Acquisition of Anelixis Therapeutics – Business Wire

IRVINE, Calif. & BOSTON--(BUSINESS WIRE)--Novus Therapeutics, Inc. (Novus) (NASDAQ: NVUS) today announced it has completed the acquisition of Anelixis Therapeutics, Inc. (Anelixis), a privately held clinical stage biotechnology company developing a next generation anti-CD40 Ligand (CD40L) antibody as a potential treatment for organ and cellular transplantation, autoimmune diseases, and neurodegenerative diseases. Concurrent with the acquisition of Anelixis, Novus entered into a definitive agreement for the sale of non-voting convertible preferred stock (the Preferred Stock) in a private placement to a group of institutional accredited investors led by BVF Partners L.P., with participation from Cormorant Asset Management, Ecor1 Capital, Logos Capital, Fidelity Management and Research Company, Adage Capital Partners L.P., Woodline Partners LP, Ridgeback Capital, Janus Henderson Investors, and Samsara BioCapital, as well as additional investors. The private placement is expected to result in gross proceeds to Novus of approximately $108 million before deducting placement agent and other offering expenses. The proceeds from the private placement will be used to fund the Companys operations, including to advance Phase 2 clinical trials of AT-1501, a humanized IgG1 anti-CD40L antibody with high affinity for CD40L, in renal transplantation, islet cell transplantation, autoimmune nephritis, and amyotrophic lateral sclerosis (ALS).

We are excited about AT-1501 and the potential to develop and commercialize the next generation anti-CD40L antibody, a well-validated target with broad therapeutic possibilities, said Keith A. Katkin, Chairman of the Board of Directors of Novus. After exploring a range of strategic options to maximize shareholder value, we believe this acquisition represents the greatest value creation opportunity for Novus stockholders, and we are confident that we have the management and scientific leadership team to fully realize this opportunity for patients in need of new treatment options.

Leadership & Organization

In addition to the strategic acquisition and private placement, Novus announced its Board of Directors has previously appointed David-Alexandre DA C. Gros, M.D. to serve as Chief Executive Officer and Director. Dr. Gros joins Novus from Imbria Pharmaceuticals Inc., where he served as Co-Founder, Chief Executive Officer and Director. Prior to Imbria, Dr. Gros was President and Chief Operating Officer of Neurocrine Biosciences, Inc., Chief Business and Principal Financial Officer of Alnylam Pharmaceuticals, Inc., and Chief Strategy Officer of Sanofi, S.A. Before Sanofi, Dr. Gros held leadership positions in healthcare investment banking at Centerview Partners, LLC, and Merrill Lynch, Pierce, Fenner & Smith, Inc., and in healthcare consulting at McKinsey & Company. Dr. Gros earned a Doctor of Medicine from Johns Hopkins University School of Medicine, a Master of Business Administration from Harvard Business School, and a Bachelor of Arts from Dartmouth College.

I am both thrilled and humbled to join the Novus management team and Board during this new phase of the companys evolution, as we prepare to initiate multiple Phase 2 trials for AT-1501 said Dr. Gros. Through this acquisition and financing, we now have the scientific, organizational and financial resources to build upon a deep historical understanding of the CD40/CD40L pathway, as well as Anelixis preclinical and Phase 1 data, to address the needs of people undergoing organ or cellular transplantation, or living with autoimmune and neurodegenerative diseases.

Joining Dr. Gros on the Novus management team and Board of Directors is Steven Perrin, Ph.D., Founder and Chief Executive Officer of Anelixis, who will take on the role of President and Chief Scientific Officer. Dr. Perrin brings 20 years of drug development experience to Novus, having held R&D positions at the Hoechst-Ariad Genomics Center, Aventis Pharmaceuticals, Inc., and Biogen Idec, Inc. Over the past decade, Dr. Perrin has worked with the ALS Therapy Development Institute to develop the worlds largest ALS drug development program, bridging preclinical and clinical programs. Dr. Perrin received a Ph.D. in biochemistry from Boston University Medical Center, where he also started his career as Associate Professor of Medicine, and a Bachelor of Science from Boston College.

The activation of CD40/CD40L signaling is critical to mediating antibody and cellular inflammatory response. We are developing antibodies to inhibit the activation of this pathway with the hope of offering new treatment modalities for people living with conditions such as autoimmune nephritis and ALS, or those requiring a potentially life-saving transplant, said Dr. Perrin. I have dedicated my career to developing better medicines for these patients and their families, and I look forward to working with the team to advance these clinical programs.

Concurrent with the acquisition, former Anelixis Chairman of the Board Walter Ogier has been appointed to the Novus Board of Directors. Mr. Ogier has more than 30 years of experience developing therapeutic medical products ranging from pharmaceuticals to medical devices, stem and immune cell therapies, and gene therapies. He has served in multiple CEO roles including Genetix Pharmaceuticals, Inc. (now bluebird bio, Inc.) and Acetylon Pharmaceuticals, Inc., which Celgene Corporation acquired in 2016. In addition to Novus, he serves as a director of Biothera Pharmaceuticals, Inc., Thetis Pharmaceuticals, LLC, and Nemucore Medical Innovations, Inc., and as Board advisor to Kodikaz Therapeutic Solutions, Inc., and ME Therapeutics, Inc.

Novus Board members will also include Keith A. Katkin, Chairman of the Board; Gary A. Lyons; and John S. McBride. The company will continue to maintain its executive offices in Irvine, Calif. and will have research and development facilities in Boston, Mass.

About the Transactions

The acquisition of Anelixis was structured as a stock-for-stock transaction whereby all of Anelixis outstanding equity interests were exchanged in a merger for a combination of shares of Novus common stock and shares of Preferred Stock. Concurrently with the acquisition of Anelixis, Novus entered into definitive agreements for a PIPE investment with existing and new investors to raise approximately $108 million in which the investors will be issued shares of Preferred Stock at a price of approximately $500 per share (or, $0.50 per share on an as-converted-to-common basis). The PIPE offering is expected to close on September 14, 2020. Subject to stockholder approval, each share of Preferred Stock will, at the option of the holder, be convertible into 1,000 shares of common stock, subject to certain beneficial ownership limitations set by each holder. The acquisition was approved by the Board of Directors of Novus and the equity holders of Anelixis.

Ladenburg Thalmann & Co. Inc. is serving as exclusive financial advisor and Gibson, Dunn & Crutcher LLP is serving as legal counsel to Novus. Goodwin Procter LLP is serving as legal counsel to Anelixis. SVB Leerink is serving as financial advisor and lead placement agent for the private placement, and Noble Life Science Partners, a division of Noble Capital Markets, Inc., is acting as co-placement agent.

Additional details are available in an updated corporate presentation that can be found online at

Webcast Details

Novus will host an audio webcast on Tuesday, September 15, 2020, at 8:30 a.m. EDT to discuss the acquisition. The live audio webcast will be accessible through a direct link and the investor section of To access via phone, please dial (833) 614-1390 (toll-free) or (914) 987-7111 (international) and provide the conference ID 4046285. Please visit the investor section of the Novus website at for the archived webcast and for more information on the acquisition.

About AT-1501

AT-1501 is a humanized IgG1 anti-CD40L antibody with high affinity for CD40L, a well-validated target with broad therapeutic potential. The CD40/CD40L pathway plays a central role in generating pro-inflammatory responses in autoimmune disease, allograft transplant rejection, and neuroinflammation. In a Phase 1 safety study of healthy volunteers and patients with ALS, AT-1501 was well tolerated at all doses tested.

About Novus Therapeutics

Novus Therapeutics, Inc. is a clinical stage biotechnology company using its expertise in targeting the CD40L pathway to develop potential treatments for people requiring an organ or cell-based transplant, and for people with autoimmune and neurodegenerative disease. Novus is headquartered in Irvine, Calif. For more information, please visit the companys website at

Follow Novus Therapeutics on social media: @Novus_Thera and LinkedIn.

Notice of Issuance of Inducement Grants

Pursuant to their employment agreements, Drs. Gros and Perrin have been awarded options to purchase a total of 18,279 and 7,857 shares of Preferred Stock, respectively, subject to time-based vesting (the "Inducement Grants"). The Inducement Grants have an exercise price of $500 per share of Preferred Stock, which is equal to the price at which the Preferred Stock is being offered and sold in the PIPE financing and represents (on an as-converted basis) a premium of approximately 30% over the last reported closing price of the Novus common stock prior to grant. The Inducement Grants have been approved by the Novus Board of Directors and the Compensation Committee of the Board of Directors. The Inducement Grants will be issued outside of the Company's stockholder-approved equity incentive plans as an inducement grant in accordance with Nasdaq Listing Rule 5635(c)(4).

Forward-Looking Statements

This press release contains forward-looking statements that involves substantial risks and uncertainties. Any statements about the companys future expectations, plans and prospects, including statements about its strategy, future operations, development of its product candidates, and other statements containing the words believes, anticipates, plans, expects, estimates, intends, predicts, projects, targets, looks forward, could, may, and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, although not all forward-looking statements include such identifying words. Forward-looking statements include, but are not limited to statements regarding: risks related to market conditions; expectations regarding the timing for the commencement of future clinical trials; expectations regarding the success of clinical trials; the rate and degree of market acceptance and clinical utility of the companys products; the companys estimates regarding expenses and cash runway; and the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the companys actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Q, annual 10-K, and other filings with the SEC, which can be found at Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise.

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Novus Therapeutics Announces Acquisition of Anelixis Therapeutics - Business Wire

Pace toddler wasn’t supposed to survive. Thanks to wonder drug, parents say he’s thriving – Pensacola News Journal

Seeing a child debilitated by illness is never easy.

When doctors tell youthere is nothing they nor you can do to help ease yourbaby's suffering, well, parents who know that type of helplessness often find it hard to describe.

We were told to take my son home and love him, becausehe probably wouldnt live past his second birthday, said Pace father Todd Hamrick. But, were way past that birthday now.

Hamricks son, Alek, was diagnosed at six months old with spinal muscular atrophy and not expected to live long enough to toddle. ButAlek, now 3, has beaten the odds and outlived that initial, bleak prognosis.

His parents attribute much of his success to his doctor, Richard Finkel, who entered Alek into a clinical trial for what they believe has been a wonder drug for their little boy, Evrysdi, which was recently approved by the Food and Drug Administration.

Whats hard to even get around is that people even bothered to research it, Todd Hamrick said. Its just a small amount of the population that has SMA. Its not like researching a blood pressure medication.

Spinal muscular atrophy, or SMA,is a genetic disorder caused by a loss of nerve cells that effect human motor function.

Essentially, those afflicted by SMA are made weak. Their muscleswaste away. In many cases, eventually, a person loses their ability to walk, to eat andevento breathe, and they die.

Aleks mother, Iwona Hamrick, is a nurse at a local hospital and wellremembers the moment she heardher sons diagnosis.

It was unimaginable. His pregnancy was normal, she said. We did genetic testing and it was negative. Unfortunately, at that time, they were not screening for his disease. So, from a healthy baby to a dying baby, you know?

The parents felt they had to travel, in more ways than one, to find the places and help that they could for their Alek.

Todd Hamrick said that he and wife decided to move from Gulf Breeze to Pace after Aleks diagnosis after feeling ostracized by many of their former acquaintances. The parents felt like some people who they used to know were made uncomfortable by their son's illness.

The area is very in-the-dark when it comes to children with issues, Todd Hamrick said. If your kids are healthy and you'rehealthy, its a great area. But, it doesnt attract the greatest talent or best and biggest facilities or endowments.

So, Alek traveled with his family to meet his future doctor, Finkel, at the Nemours Children's Hospital in Orlando.

Alekwas first treated with a gene therapy drug, and he made some response with that, Finkel told the News Journal. But more recently, he started on a second drug, which seems to be having an enhanced effect, I must say.

Finkel, an expert in the field of pediatric neurologic disorders, left Florida in March for a position leading the new Center for Experimental Neurotherapeutics at St. Jude Childrens Research Hospital in Memphis, Tennessee. Buthe has continued to monitor Aleks progress via video.

Both of the drugs he has received, the gene therapy and this new drug, Evrysdi, are designed to increase a certain protein in Aleks body that is deficient because of his genetic disorder, he explained. But they do it in different ways. The potential advantage of his new drug, Evrysdi because its an oral drug; you take it by mouth it goes into the stomach and into the bloodstream, and from there, it goes to all the tissues of the body.

And, we think that there is an enhanced effect, becauseit gets into the muscle tissue, Finkel continued. "These are very, very, early daysin trying to make assessments, soI dont want to say that we can come to any kind of conclusions yet."

However the cutting-edge drug works doesnt matter to a mother, whos just glad that it isworking.

It gave us hope. Thats for sure, Iwona Hamrick said. Becausewe felt helpless.

Since Alek started his new treatment last November, his strength has increased tremendously.

His muscle tone got better, Iwona Hamrick said. He is much stronger in the upper body, so much so, he is pushing his little wheelchair.

Alek can now cruise around his Pace home in an extraordinarily lite-weight wheelchair designed by a Swedish inventor who alsohas a child with SMA.

Alek had a lot of trouble before starting this medication even pushing it, Iwona Hamrick said. Sonowhe is just rolling around the house. Also, he is barring more weight on his legs.

Recently, Alek has started to be able to walk in a pool a huge milestone for the toddler.

But some worries remain the same.

Every day, Alek must use a type of breathing machine.

Its a cough assist machine, Todd Hamrick explained. We use it two times a day, when hes healthy. Becausewhere we can just clear our throats when we cough, he doesnt have that strength no lung strength.

Alek attends physical therapy, aqua-therapy,hippotherapy, occupational therapy and speech therapy sessions every week to try and ensure he remains healthy and continues to properly develop.

My worries have changed a lot, Todd Hamrick said. I used to worry my child was going to die. Now, Im worrying about if other kids will bully him at school.

"But that'sa great worry to have," he continued. "Compared to how it use to be, getting picked on is a great thing to worry about.

Colin Warren-Hicks can be reached at or 850-435-8680.

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Pace toddler wasn't supposed to survive. Thanks to wonder drug, parents say he's thriving - Pensacola News Journal